Category Archives: Regenerative Medicine

UCL has an impressive track record of biomedical translational discovery founded upon academic excellence combined with a close relationship with patients and clinicians. This pipeline of innovation is supported through our professional infrastructure linking UCL to our partner hospitals, exemplified by our three NIHR Biomedical Research Centres (UCL Hospital, Moorfields Eye Hospital and Great Ormond Street Hospital for Children BRCs).

The portfolio is typified by, IP generation, licensing and commercialisation and multiple types of partnerships with not-for-profit companies, creation of social ventures and collaborative research with industrial partners, all aimed to address the most pressing patient, hospital and societal needs.

The research translational ecosystem has changed significantly over the past 10 years. Pharma and biotech industries have reorganised this, cutting back on internal research to free-up resource to fund more open innovation and collaborative science. This has led to fragmentation of the delivery of innovation with an increase in the number of SMEs and growth of CROs to fill the gap.

The governments translational strategy and subsequent evolution of the Industrial Strategy has seen the addition of significant investment to incentivise academics and clinicians to participate in biomedical translation which has transformed the sector.

Below we describe some of the important aspects we consider vital for successful biomedical translation that have evolved within the UCL innovation ecosystem over the past 10 years.

UCL and its partners have put in place flexible, professional and responsive structures that are highly networked and aligned to deliver a step-fold change in delivering biomedical translational impact (Figure 1).

In 2010, taking advantage of downsizing activities in large pharmaceutical companies, UCL established the Translational Research Office (TRO) with senior experienced scientists from industry. Embedded within the universitys School of Life and Medical Sciences, the team provide their complimentary therapeutic development know-how to the academics expertise in their fields of research.

The TRO adopt a life-cycle management approach and establish with the academic the appropriate team to deliver the project for societal impact (e.g. clinical, regulatory, manufacturing, toxicology, PK/PD, commercial etc.).

UCLB business managers are key to realising the commercial possibilities of the academics research and ideas, and are essential to delivering the potential to make a real and positive impact on peoples lives. This coordinated approach, uniting the right mix of knowledge and expertise in support of a shared biomedical translational portfolio has allowed the acceleration of research and the ignition of innovation (Figure 2).

Access to funding is critical to accelerating the translation of projects. Within UCL there are several funds available to researchers that can help them advance their projects:

UCL have taken steps to build upon and accelerate the strong academic drive to translate their novel biomedical science. This is being maximised by coordinating professional support with the interested research community around a particular therapeutic modality and sharing knowledge through the Therapeutic Innovation Networks (TINs).

These multidisciplinary networks have been established in small molecules, cell, gene and regenerative medicine, biologics, repurposing, devices and diagnostics. Supported through a Wellcome Trust Translational Partnership Award, TINs aim to increase knowledge and understanding of translation and patenting, and equip researchers with the skills they need to collaborate with commercial partners.

Translation is a team sport that requires multiple players to be aligned and pulling in the same direction. UCL and its partner hospitals strategy over the past 10 years has enabled the acceleration of biomedical therapies delivering patient impact. The significant commercialisation of its world-leading, world-changing technologies and innovations into spinout companies has collectively raised in excess of 1 billion investment (Figure 2) making UCL the go-to place for organisations looking to partner with academia and hospitals in research, education, innovation and industrial engagement along the entire value chain of therapeutic development.

UCL and its partner hospitals have a well-established innovation ecosystem helping to develop entrepreneurial researchers and drive a pipeline of marketable therapeutic innovations. As we look forward to the next ten years, we will continue to evolve our structures to provide the necessary support to our researchers to advance their amazing science for patient benefit.

Please note: This is a commercial profile

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Partnerships are key to biomedical translational success - Open Access Government

ORLANDO, Fla.--(BUSINESS WIRE)--Amnion of Florida, a leading provider of alternative medicine utilizing cryopreserved placental cell allograft for advanced bioactive facial rejuvenation, is pleased to announce a partnership to enhance patient outcomes for joint pain and anti-aging therapies.

Amnion announces a partnership with Care First Medical Group, Inc. to improve patients experience with managed medical care and provide non-pharmaceutical solutions for joint repair/pain management and anti-aging. The goals of the collaboration are to provide alternatives to medical care and to help patients seeking to eliminate chronic joint pain, look younger, and take a more active role in mapping their medical care to avoid illness in the future.

Sabriya Rogers, President of Care First, states, Our Board-Certified primary care providers specialize in managed health, thus patients can take a proactive approach in deciding the course of their care, which yields a lower cost to patients, while keeping families together, and maintaining a high degree of medical care.

The partnership will provide more location options for patients. With offices throughout greater Orlando, Sanford, Leesburg, Palm Coast, and Longwood, patients will find a location nearby that fits busy schedules.

Our team of medical professionals and aestheticians at Amnion are excited to partner with Care First to provide enhanced medical care and rejuvenation services at our partnership locations, said Eusebio Coterillo, President of Amnion.

In a constantly changing field, Amnion of Florida, under the guidance of an on-site medical staff, provides the highest level of quality products and procedures in regenerative medicine. They offer cutting edge treatments that are shown to be effective, use FDA cleared or registered products, and are widely published and peer endorsed. The collaboration with Care First will bring added benefits such as managed care, urgent care, and programmed care for long term issues such as high blood pressure, diabetes, high cholesterol, asthma, and allergies in a full-service clinical setting.

More about Amnion of Florida

Amnion of Florida, based in Central Florida, is a leading provider of alternative medicine using cryopreserved placental cell transplants or allografts, processed from donated cellular birth tissue, which are natural alternatives to autologous regenerative medicine products. The primary function of our allogeneic regenerative treatments is to promote soft tissue joint/skin repair and regeneration mediated by growth factors and cells naturally found in placental tissue. These treatments have shown safety and efficacy in treating a variety of ailments including osteoarthritis, chronic ulcerative wounds, joint pain, skin rejuvenation, hair restoration, urinary incontinence, and ED. Learn more at http://www.amnion.us.

More about Care First Medical Group, Inc.

Care First, based in Central Florida, is focused on providing affordable healthcare for primary and urgent care needs through prevention, patient education, with the personal touch to improve communication with the patient, and patients overall health. Our vision is to transform the health care experience through a culture of caring, quality, safety, service, innovation, and excellence. Our goal is to be recognized by employees, physicians, patients and families, volunteers, and the community as a company that delivers results through our proactive approach to medicine. Learn more or schedule an appointment at (407) 449-0632.

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Amnion of Florida Announces Greater Geographic Reach for Their Regenerative Health Services by Partnering With Care First Medical Group - Business...

The recent report added by Verified Market Research gives a detailed account of the drivers and restraints in the Global Regenerative Medicine market. The research report, titled [Global Regenerative Medicine Market Size and Forecast to 2026] presents a comprehensive take on the overall market. Analysts have carefully evaluated the milestones achieved by the global Regenerative Medicine market and the current trends that are likely to shape its future. Primary and secondary research methodologies have been used to put together an exhaustive report on the subject. Analysts have offered unbiased outlook on the global Regenerative Medicine market to guide clients toward a well-informed business decision.

Global Regenerative Medicine Market was valued at USD 19.10 Billion in 2018 and is expected to witness a growth of 22.72% from 2019-2026 and reach USD 98.10 Billion by 2026.

The comprehensive research report has used Porters five forces analysis and SWOT analysis to give the readers a fair idea of the direction the global Regenerative Medicine market is expected to take. The Porters five forces analysis highlights the intensity of the competitive rivalry while the SWOT analysis focuses on explaining strengths, weaknesses, opportunities, and threats present in the global Regenerative Medicine market. The research report gives an in-depth explanation of the trends and consumer behavior pattern that are likely to govern the evolution of the global Regenerative Medicine market.

The following Companies as the Key Players in the Global Regenerative Medicine Market Research Report:

Regions Covered in the Global Regenerative Medicine Market:

Europe (Germany, Russia, UK, Italy, Turkey, France, etc.)

The Middle East and Africa (GCC Countries and Egypt)

North America (United States, Mexico, and Canada)

South America (Brazil etc.)

Asia-Pacific (China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

The scope of the Report:

The research report on global Regenerative Medicine market includes segmentation on the basis of technology, application, end users, and region. Each segmentation is a chapter, which explains relevant components. The chapters include graphs to explain the year-on-year progress and the segment-specific drivers and restraints. In addition, the report also provides the government outlooks within the regional markets that are impacting the global Regenerative Medicine market.

Lastly, Verified Market Researchs report on Regenerative Medicine market includes a detailed chapter on the company profiles. This chapter studies the key players in the global Regenerative Medicine market. It mentions the key products and services of the companies along with an explanation of the strategic initiatives. An overall analysis of the strategic initiatives of the companies indicates the trends they are likely to follow, their research and development statuses, and their financial outlooks. The report intends to give the readers a comprehensive point of view about the direction the global Regenerative Medicine market is expected to take.

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Table of Content

1 Introduction of Regenerative Medicine Market

1.1 Overview of the Market 1.2 Scope of Report 1.3 Assumptions

2 Executive Summary

3 Research Methodology of Verified Market Research

3.1 Data Mining 3.2 Validation 3.3 Primary Interviews 3.4 List of Data Sources

4 Regenerative Medicine Market Outlook

4.1 Overview 4.2 Market Dynamics 4.2.1 Drivers 4.2.2 Restraints 4.2.3 Opportunities 4.3 Porters Five Force Model 4.4 Value Chain Analysis

5 Regenerative Medicine Market, By Deployment Model

5.1 Overview

6 Regenerative Medicine Market, By Solution

6.1 Overview

7 Regenerative Medicine Market, By Vertical

7.1 Overview

8 Regenerative Medicine Market, By Geography

8.1 Overview 8.2 North America 8.2.1 U.S. 8.2.2 Canada 8.2.3 Mexico 8.3 Europe 8.3.1 Germany 8.3.2 U.K. 8.3.3 France 8.3.4 Rest of Europe 8.4 Asia Pacific 8.4.1 China 8.4.2 Japan 8.4.3 India 8.4.4 Rest of Asia Pacific 8.5 Rest of the World 8.5.1 Latin America 8.5.2 Middle East

9 Regenerative Medicine Market Competitive Landscape

9.1 Overview 9.2 Company Market Ranking 9.3 Key Development Strategies

10 Company Profiles

10.1.1 Overview 10.1.2 Financial Performance 10.1.3 Product Outlook 10.1.4 Key Developments

11 Appendix

11.1 Related Research

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Regenerative Medicine Market Segmentation, Top Companies, Applications, Comprehensive Research Report and Forecast to 2026 - Market Research Sheets

At the 200-year mark, some institutions would be content to rest on their laurels. But not Indiana University. As their bicentennial celebrations nears, the Hoosiers have dedicated themselves to a tackling what they are calling Grand Challenges, a suite of economic, social and environmental problems plaguing Indiana and the world. These challenges are marked by their impressive scale, immense impact, driven commitment to solutions, and a spirit of interdisciplinary collaboration.

One of the Grand Challenges is the Precision Health Initiative, which aims to match patients more closely and carefully with treatments that meet their individual needs. According to Anatha Shekhar, the executive associate dean of research affairs at the Indiana University School of Medicine, the initiative derives that precision by taking a decidedly holistic look at a patient's health.

"What we're trying to do here is to have a comprehensive approach to understanding a person's health, taking into account their genetics, their environment, their behavior, their culture, and to understand the diseases that are caused by these factors," Shekar explains. "We're particularly focusing on five major serious illnesses that currently have no good treatments and that are uniformly lethal or disabling."

Those five diseases include three types of cancer: triple-negative breast cancer, multiple myeloma, and pediatric sarcoma. They're also targeting diabetes, in particular gestational diabetes affecting pregnant women, as well as Alzheimer's disease.

The Precision Health Initiative was the first project to receive Grand Challenges funding. A truly transformative undertaking, Indiana is pulling together researchers and physicians from across the university ecosystem to create new approaches to prevention, treatment and health care delivery.

"The Precision Health Initiative is trying to just marshal the great resources we have here, all the scientific minds, all the technology, to attack big, bold problems," says Aaron Carroll, professor of pediatrics and associate dean for research mentoring at the IU School of Medicine.

The project is funneling $120 million into six different major scientific pillars: Genomic Medicine; Cell, Gene & Immune Therapy; Regenerative Medicine & Engineering; Psychosocial, Behavioral & Ethics; Data & Informatics; and Chemical Biology & Biotherapeutics.

Carroll likens it all to a moonshot. Where research is normally understood to move slowly, taking tiny steps towards a solution, Indiana seeks great strides founded in unfettered access to both real and mental capital. And, as the only medical school in the state, as well as a regional and national research leader, their charge is an imperative. "It's certainly very Hoosier. I think the idea that we're going to take our local expertise and really try to band together and cross boundaries to try to make a big difference, it's, it's very Indiana," Carroll says.

Clinically speaking, the Precision Health Initiative is not just seeking to draw down instances of the maladies their targeting. Rather, they seek wholesale cures for the cancers, and robust, workable preventative measures for diabetes and Alzheimer's disease.

Their patient-focused approach is already being taught to physicians-in-training at the IU School of Medicine. The importance of factors such as genetic and family medical history, sleep and eating habits, exercise levels and overall mental health is emphasized alongside traditional testing in making a diagnosis.

In the lab, Indiana researchers are matching that holistic approach to patient health with an equally vibrant understanding of how diseases operate. They're working towards a more-perfect model of exactly how cancers evolve and how they become resistant to certain treatments. Concurrently, pharmaceutical scientists are making gains in drug discovery for the treatment of Alzheimer's.

Shekhar says that the feeling among the Precision Health Initiative team is that the work being done now is serving as inspiration for others to "think big and to go for the fences rather than do incremental work."

There are, though, factors limiting the speed at which the team can make new discoveries. Key among them is the need for an expansion in data analysis and management as well as advances need in AI to help researchers make sense of extremely large data sets.

One such set derives from the All In project, which is under the Precision Health Initiative umbrella. Researchers are asking people from across the state and all walks of life to share with them a blood sample as well as specific, yet anonymous health data. This will aid immensely in their understanding of how and why diseases flourish and progress in various populations.

That particular project is indicative of the larger initiative, says Shekhar, echoing Carroll's sentiment that Indiana is uniquely positioned to helm such an audacious undertaking.

"I think Indiana is unique in that first of all, we have the largest medical school in the country, that is a state wide medical school with nine campuses," he says. "We have the largest health system in the state. We serve about two million people every year for their health needs. So, we have gone all the way from engaging large scientific teams, recruiting nearly 50 new scientists to Indiana University to work on this, to engaging nearly hundred thousand people from the community. It's an initiative that couldn't happen in very many places except in Indiana."

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Inside Indiana's Precision Health Initiative: BTN LiveBIG - Big Ten Network

Cell and Advanced Therapies Supply Chain Management Market: Focus on Technological Solutions, 2019-2030report features an extensive study of the growing supply chain management software solutions market.

The focus of this study is on software systems, including cell orchestration platforms (COP), enterprise manufacturing systems (EMS), inventory management systems (IMS), laboratory information management systems (LIMS), logistics management systems (LMS), patient management systems (PMS), quality management systems (QMS), tracking and tracing software (TTS), and other such platforms that are being used to improve / optimize various supply chain-related processes of cell and advanced therapies.

One of the key objectives of the report was to understand the primary growth drivers and estimate the future size of the supply chain management software solutions market. Based on multiple parameters, such as number of cell and advanced therapies under development, expected pricing, likely adoption rates, and potential cost saving opportunities from different software systems, we have developed informed estimates of the evolution of the market, over the period 2019-2030.

In addition, we have provided the likely distribution of the current and forecasted opportunity across:

Advanced therapy medicinal products, such as cell and gene therapies, have revolutionized healthcare practices. The introduction of such treatment options has led to a paradigm shift in drug development, production and consumption. Moreover, such therapies have actually enabled healthcare providers to treat several difficult-to-treat clinical conditions.

In the past two decades, more than 30 such therapy products have been approved; recent approvals include Zolgensma (2019), RECELL System (2018), AmnioFix (2018), EpiFix (2018), EpiBurn (2018), Alofisel (2018), LUXTURNA (2017), Yescarta (2017), and Kymriah (2017). Further, according to a report published by The Alliance for Regenerative Medicine in 2019, more than 1,000 clinical trials are being conducted across the globe by over 900 companies.

In 2018, around USD 13 billion was invested in this domain, representing a 73% increase in capital investments in this domain, compared to the previous year. It is worth highlighting that, based on an assessment of the current pipeline of cell therapies and the historical clinical success of such products, it is likely that around 10-20 advanced therapies are approved by the US FDA each year, till 2025.

The commercial success of cell and advanced therapies is not only tied to whether they are capable of offering the desired therapeutic benefits, but also on whether the developers are able to effectively address all supply chain requirements. The advanced therapy medicinal products supply chain is relatively more complex compared to the conventional pharmaceutical supply chain. As a result, there are a number of risks, such as possible operational inefficiencies, capacity scheduling concerns, process delays leading to capital losses, and deliverable tracking-related issues, which need to be taken into consideration by therapy developers.

This has generated a need for bespoke technological solutions, which can be integrated into existing processes to enable the engaged stakeholders to oversee and manage the various aspects of the cell and advanced therapies supply chain, in compliance to global regulatory standards. Over the years, several innovative, software-enabled systems, offering supply chain orchestration and needle-to-needle traceability, have been developed.

The market has also recently witnessed the establishment of numerous partnerships, most of which are agreements between therapy developers and software solutions providers. Further, given the growing demand for cost-effective personalized medicinal products, and a myriad of other benefits of implementing such software solutions, the niche market is poised to grow significantly in the foreseen future.

Amongst other elements, the report features:

In order to account for the uncertainties associated with some of the key parameters and to add robustness to our model, we have provided three market forecast scenarios portraying the conservative, base and optimistic tracks of the industry's evolution.

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Supply Chain Management Software Solutions Market Will Grow Significantly in Coming Years - Supply and Demand Chain Executive

WASHINGTON, Dec. 20, 2019 (GLOBE NEWSWIRE) -- via NEWMEDIAWIRE -- The Alliance for Regenerative Medicine (ARM), the international advocacy organization for the cell and gene therapy and broader regenerative medicine sector, today released the initial slate of presenting companies at the 2020 Cell & Gene Meeting on the Mediterranean. The event will be held April 15-17, 2020 in Barcelona, Spain.

The event, modeled after ARMs highly successful Cell & Gene Meeting on the Mesa, is expected to attract more than 500 attendees, including senior executives from leading cell therapy, gene therapy, and tissue engineering companies worldwide, large pharma and biotech representatives, institutional investors, academic research institutions, patient foundations, disease philanthropies, and members of the life science media community.

The second annual Cell & Gene Meeting on the Mediterranean will feature presentations by 50+ leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies.

The initial slate of 2020 presenting companies includes: Adaptimmune, AGTC, Ambys Medicines, American Gene Technologies, AskBio, Aspect Biosystems, Atara, Autolus Therapeutics, Avectas, AVROBIO, Axovant Gene Therapies, bluebird bio, Bone Therapeutics, Caribou Biosciences, Celavie Biosciences, Cellatoz Therapeutics, CEVEC, Cynata Therapeutics, Flexion Therapeutics, Fraunhofer IZI, GenSight Biologics, Healios, Iovance Biotherapeutics, Kiadis Pharma, Kytopen, LogicBio Therapeutics, MeiraGTx, Minerva Biotechnologies, MolMed, Novadip Biosciences, Orchard Therapeutics, Oxford Biomedica, PDC*line Pharma, Precision BioSciences, Promethera Biosciences, PTC Therapeutics, Recombinetics, REGENXBIO, ReNeuron, Rexgenero, Sangamo, SmartPharm Therapeutics, Standards Coordinating Body for Regenerative Medicine, Theradaptive, ThermoGenesis, Tmunity Therapeutics, Ultragenyx Pharmaceutical, VERIGRAFT, and Zelluna Immunotherapy.

Additional event details will be updated regularly on the conference website http://www.meetingonthemed.com.

Registration is complimentary for investors and credentialed members of the media. To learn more and to register, please visitwww.meetingonthemed.com. For members of the media interested in attending, please contact Kaitlyn Donaldson Dupont at kdonaldson@alliancerm.org.

For interested organizations looking to increase exposure to this fields top decision-makers via sponsorship, please contact Kelly McWhinney at kmcwhinney@alliancerm.org for additional information.

About The Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 350 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visithttp://www.alliancerm.org.

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The Alliance for Regenerative Medicine Releases Initial Slate of Presenting Companies at the 2020 Cell & Gene Meeting on the Mediterranean - Yahoo...

Croatian scientists from the St. Catherine Specialty Hospital published an extremely important scientific paper in the Genes scientific journal, showing that the injection of the stromal and the mesenchymal stem cells into the knee joint shows long-term effects when measured 24 months after application.

The paper, which you can read here (full text of the paper is available if the reading of highly technical and scientific papers is your thing) is called "A 24-Month Follow-up Study of the Effect of Intra-Articular Injection of Autologous Microfragmented Tissue on Proteoglycan Synthesis in Patients with Knee Osteoarthritis. It's a multicentric project, with the goal to confirm the effect of micro-fragmented fat tissue (stromal vascular fraction from microfragmented lipoaspirate, so-called SVF) intra-articular injection 24 months after application, in the patients suffering from osteoarthritis (OA). The project head and the corresponding author of the paper was professor Dragan Primorac PhD, and the other authors are St. Catherine's Igor Bori, Damir Hudetz, Eduard Rod, eljko Jele, Andrea Skelin, Mihovil Pleko, and their partners from other Croatian scientific institutions Trpimir Vrdoljak, Ozren Polaek. Irena Trbojevi-Akmai and Gordan Lauc.

The results of this study suggest that the mesenchymal stromal and the mesenchymal stem cells separated from the microfragmented fat tissue lead to the increase of the key molecules of cartilage (the so-called glycosaminoglycans (GAGs)) two years after the application within the joint. Although the numbers for 24 months after the application were somewhat lower than when measured 12 months after the application, in over 50% of the subjects (52 per cent) they were higher than before the treatment. The glycosaminoglycans (GAG) content in cartilage by means of delayed gadolinium (Gd)-enhanced magnetic resonance imaging of cartilage (dGEMRIC), as well as the clinical outcome on observed level of GAG using standard orthopaedic physical examination

Lucija Zeni and Denis Polanec from the Srebrnjak Children's Hospital helped the team in using the methods of immunophenotyping and flow cytometry to determine the types and the content of the MSC, determining the dominant populations of cells. At the same time, while examining the clinical results of the treatment of the knee with the autologous micro-fragmented fat tissue it was determined that 85 per cent of the patients report the significant improvement, as confirmed by the standard orthopaedic tests, such as Knee Injury and Osteoarthritis Score (KOOS) and Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC), as well as pain intensity measurement - VAS scale.

At the same time, the team at St. Catherine's hospital wanted to report that the leading scientific magazine Nature published a chapter on "The Future of Cartilage Repair in the book they published called "Personalized medicine in Healthcare Systems", in which the doctors from their hospital participated as authors. They've argued that the production of the bioactive molecules increases the improvement of a number of measurable parameters in patients, and because of that specific effect they would like the "Mesenchymal Stem Cells" to be renamed the "Medicinal SIgnaling Cells". The new findings in the modern regenerative medicine and the available methods of therapy, performed at the St. Catherine's Specialty Hospital give new hope to the patient, provide the newest breakthroughs in the treatment of this disease, but also position the Croatian health system powerfully worldwide.

Osteoarthritis is one of the most common health problems in the world with the increasingly ageing population, and some estimated say that currently, over 600 million people suffer from it. The treatment has been based on relieving the symptoms and implanting the endoprosthesis when it was determined that the cartilage can not be salvaged.

Prof. Dragan Primorac, PhD said that the results published in the Gened magazine and the findings that were published changed some existing paradigms, and show the way towards a better understanding of the biology and the therapeutic effect of the treatment of the osteoarthritis with autologous stromal and stem cells present within the microfragmented fat tissue. In osteoarthritis and in numerous other diseases, the future of medicine will be based on the integration of the principles of personalized and regenerative medicine into the clinical practice. I am happy that the Croatian experts are allowed once more to prove their global excellence, and I'm especially happy that the results of our research have a great impact on the treatment of the patients suffering from osteoarthritis. It is clear that our results have an extraordinary significance in the development of the new diagnostic, therapeutic and prognostic algorithms related to osteoarthritis.

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St. Catherine Hospital Scientists Confirm Long-Term Benefits of Stem Cells Therapy - Total Croatia News

MEDFORD/SOMERVILLE, Mass. (December 16, 2019) Researchers led by engineers at Tufts University have developed a novel, significantly more efficient fabrication method for silk that allows them to heat and mold the material into solid forms for a wide range of applications, including medical devices. The end products have superior strength compared to other materials, have physical properties that can be tuned for specific needs, and can be functionally modified with bioactive molecules, such as antibiotics and enzymes. The thermal modeling of silk, described in Nature Materials, overcomes several hurdles to enable manufacturing flexibility common to many plastics.

We and others have explored the development of many silk-based devices over the years using solution-based manufacturing, said David Kaplan, Stern Family Professor of Engineering at the Tufts University School of Engineering and corresponding author of the study.But this new solid-state manufacturing approach can significantly cut the time and cost of producing many of them and offer even greater flexibility in their form and properties. Further, this new approach avoids the complications with solution-based supply chains for the silk protein, which should facilitate scale up in manufacturing.

Silk is a natural protein-based biopolymer that has long been recognized for its superior mechanical properties in fiber and textile form, producing durable fabrics and used in clinical sutures for thousands of years. Over the past 65 years, scientists have devised ways to break down the fibers and reconstitute the silk protein, called fibroin, into gels, films, sponges and other materials for applications that range from electronics to orthopedic screws, and devices for drug delivery, tissue engineering, and regenerative medicine. However, breaking down and reconstituting fibroin requires a number of complex steps. Additionally, the instability of the protein in aqueous soluble form sets limits on storage and supply chain requirements, which in turn impacts the range and properties of materials that can be created.

The researchers reported that they have overcome these limitations by developing a method for solid-state thermal processing of silk, resulting in the molding of the protein polymer directly into bulk parts and devices with tunable properties. The new method similar to a common practice in plastics manufacturing - involves the fabrication of nanostructured pellets with diameters from 30 nanometers to 1 micrometer that are produced by freeze drying an aqueous silk fibroin solution. The nanopellets are then heated from 97 to 145 degrees Celsius under pressure, when they begin to fuse. The pleated pattern structure of the silk protein chains become more amorphous, and the fused pellets form bulk materials that are not only stronger than the solution-derived silk materials but also superior to many natural materials such as wood and other synthetic plastics, according to the researchers. The pellets are an excellent starting material since they are stable over long periods and thus can be shipped to manufacturing sites without the requirement for bulk water, resulting in significant savings in time and cost.

The properties of the heat molded silk, such as flexibility, tensile and compression strength, can be tuned to specific ranges by altering the conditions in the molding process, such as temperature and pressure, while the bulk materials can be further machined into devices, such as bone screws and ear tubes, or imprinted with patterns during or after the initial molding. Adding molecules such as enzymes, antibiotics or other chemical dopants allows for the modification of the bulk materials into functional composites.

To demonstrate applications, the researchers tested the bone screws developed with solid state molding in vivo and found they showed biocompatibility as implanted devices, where they supported the formation of new bone structure on the screw surfaces without inflammation. The silk screws were also able to resorb as they were being replaced by bone tissue. Resorption rate can be tuned by preparing screws at different temperatures, ranging from 97 degrees to 145 degrees Celsius, which alters the crystallinity of the bulk material, and therefore its ability to absorb water.

The researchers also manufactured ear tubes -- devices used to help drain infected ear canals -- doped with a protease, which breaks down the silk polymer to accelerate degradation as needed after the tube has served its function.

The thermal molding process is made possible because the amorphous silk has a well-defined melting point at 97 degrees Celsius, which earlier solution-based preparations did not exhibit, said Chengchen Guo, post-doctoral scholar in the Kaplan lab and co-first author of the study. That gives us a lot of control over the structural and mechanical properties of what we make. Chunmei Li, Tufts research assistant professor who teamed up with Guo as first author, added that the starting material the nanopellets are also very stable and can be stored over long periods. These are significant advances that can improve the application and scalability of silk product manufacturing.

This work was supported by grants from the U.S. National Institutes of Health (NIH) (R01AR068048, R01DE016525) and the Air Force Office of Scientific Research (FA9550-17-1-0333). Its contents are solely the responsibility of the authors and do not necessarily represent the official views of the NIH.

Guo C, Li, C., Vu,H.V., Hanna, P., Lechtig, A., Qiu, Y., Mu, X., Ling, S., Nazarian, A., Lins, S., and Kaplan, D.L. "Thermoplastic molding of regenerated silk. Nature Materials (16 Dec 2019), DOI: 10.1038/s41563-019-0560-8

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About Tufts University

Tufts University, located on campuses in Boston, Medford/Somerville and Grafton, Massachusetts, and in Talloires, France, is recognized among the premier research universities in the United States. Tufts enjoys a global reputation for academic excellence and for the preparation of students as leaders in a wide range of professions. A growing number of innovative teaching and research initiatives span all Tufts campuses, and collaboration among the faculty and students in the undergraduate, graduate and professional programs across the university's schools is widely encouraged.

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Researchers mold silk into wide range of products - Tufts Now

The unpredictable nature of OFF episodes can be extremely challenging and disruptive to the daily lives of people living with Parkinsons disease as well as their care partners, said Antony Loebel, M.D., President and Chief Executive Officer at Sunovion. We look forward to working with the FDA over the remaining review period.

People with Parkinson's have shared that OFF episodes can be disruptive and hamper their quality of life. New treatments could mean greater symptom control for more people, improving their ability and confidence to navigate daily life with the disease, said Todd Sherer, PhD, CEO of The Michael J. Fox Foundation for Parkinsons Research.

Apomorphine sublingual film is being developed as a fast-acting medicine for the on-demand treatment of all types of motor OFF episodes, including morning OFF, unpredictable OFF, delayed ON/partial ON and end-of-dose wearing OFF. OFF episodes can cause severe disruption for someone living with PD, often causing a major disturbance in a persons ability to maintain everyday activities. OFF episodes may worsen in frequency and severity over the course of the illness.1 While OFF episodes are experienced by 40 to 60 percent of people with PD, there are limited treatment options available to treat OFF episodes when they occur. 1

About Apomorphine Sublingual Film Apomorphine sublingual film (APL-130277), a novel formulation of apomorphine, a dopamine agonist, is being developed as a fast-acting on-demand treatment of OFF episodes associated with Parkinsons disease (PD). Apomorphine sublingual film is designed to offer a potential option that may be used to treat OFF episodes associated with PD up to five times throughout the day. It may help people with PD rapidly convert from the OFF to the ON state. Results of the pivotal Phase 3 study (CTH-300) of apomorphine sublingual film were recently published in Lancet Neurology. Apomorphine sublingual film has not been approved by the U.S. Food and Drug Administration (FDA). In October 2016, Sunovion acquired Cynapsus Therapeutics Inc. (Canadian Specialty Central Nervous System Biotechnology Company), along with its product candidate APL-130277. Two early-stage APL-130277 trials were funded in part by The Michael J. Fox Foundation.

About Parkinsons Disease and OFF Episodes By 2030 it is estimated that 1.2 million people in the U.S. and an estimated 10 million people worldwide will be living with Parkinsons disease (PD).2 PD is a chronic, progressive neurodegenerative disease characterized by motor symptoms, including tremor at rest, rigidity and impaired movement, as well as significant non-motor symptoms, including cognitive impairment and mood disorders. It is the second most common neurodegenerative disease behind Alzheimers disease, and the prevalence of PD is increasing with the aging of the population. OFF episodes are the worsening or re-emergence of PD symptoms (motor and non-motor) otherwise controlled by medications. These episodes may disrupt a persons ability to perform everyday activities and may be burdensome for patients, family and care partners. OFF episodes are experienced by 40 to 60 percent of people with PD and may worsen in frequency and severity over the course of the illness. 1

About Sunovion Pharmaceuticals Inc. (Sunovion) Sunovion is a global biopharmaceutical company focused on the innovative application of science and medicine to help people with serious medical conditions. Sunovions vision is to lead the way to a healthier world. The companys spirit of innovation is driven by the conviction that scientific excellence paired with meaningful advocacy and relevant education can improve lives. With patients at the center of everything it does, Sunovion has charted new paths to life-transforming treatments that reflect ongoing investments in research and development and an unwavering commitment to support people with psychiatric, neurological and respiratory conditions.

Headquartered in Marlborough, Mass., Sunovion is an indirect, wholly-owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. Sunovion Pharmaceuticals Europe Ltd., based in London, England, and Sunovion Pharmaceuticals Canada Inc., based in Mississauga, Ontario, are wholly-owned direct subsidiaries of Sunovion Pharmaceuticals Inc. Additional information can be found on the companys websites: http://www.sunovion.com, http://www.sunovion.eu and http://www.sunovion.ca. Connect with Sunovion on Twitter, LinkedIn, Facebook and YouTube.

About Sumitomo Dainippon Pharma Co., Ltd. Sumitomo Dainippon Pharma is among the top-ten listed pharmaceutical companies in Japan, operating globally in major pharmaceutical markets, including Japan, the U.S., China and the European Union. Sumitomo Dainippon Pharma aims to create innovative pharmaceutical products in the Psychiatry & Neurology area, the Oncology area and Regenerative medicine/Cell therapy field, which have been designated as the focus therapeutic areas. Sumitomo Dainippon Pharma is based on the merger in 2005 between Dainippon Pharmaceutical Co., Ltd., and Sumitomo Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more than 6,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma is available through its corporate website at https://www.ds-pharma.com.

SUNOVION is a registered trademark of Sumitomo Dainippon Pharma Co., Ltd.

Sunovion Pharmaceuticals Inc. is a U.S. subsidiary of Sumitomo Dainippon Pharma Co., Ltd. 2019 Sunovion Pharmaceuticals Inc. All rights reserved.

For a copy of this release, visit Sunovions website at http://www.sunovion.com

References

1 Denny, A. Motor fluctuations in Parkinsons disease. Journal of the Neurological Sciences. May 1999, Vol. 165, Issue 1, p. 18-23, table 3. Available online: http://www.jns-journal.com/article/S0022-510X(99)00052-0/fulltext. Accessed February 2017.2 Parkinsons Disease Foundation Website: https://www.parkinson.org/about-us/Press-Room/Press-Releases/New-Study-Shows-Over-1-Million-People-in-the-United-States-Estimated-to-be-Living-with-Parkinsons-Disease-by-2030. Accessed December 2019.

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Sunovion Announces FDA Filing Acceptance of Resubmitted New Drug Application for Apomorphine Sublingual Film - BioSpace

Friday, December 13, 2019

Following up on our first post in this year-end series that discussed medical device regulatory activities at the Food and Drug Administration (FDA), the Mintz FDA teams second year-end post will provide an overview of 2019 with a focus on the drug, biologic, and regenerative medicine programs at the agency. In many ways, the past year could be called a business as usual year for the FDAs drugs and biologics centers in that they continued to make progress on all of large-scale programs and priorities initiated by former-Commissioner Scott Gottlieb, who left the agency in April. FDA has been under the leadership of an Acting Commissioner since that time, although Texas radiation oncologist Dr. Stephen Hahn will be taking the reins soon following his confirmation by the full Senate in a 72-18 vote on December 12, 2019. (The Senate HELP Committee advanced the nominee on December 3, 2019; see our blog post just prior to that committee vote here.)

At the same time, however, the final months of 2019 have exposed several challenges for various FDA programs that operate under the extensive drug and biologic authorities contained in the Food Drug & Cosmetic Act (FD&C Act) and the Public Health Service Act (PHS Act), respectively. The agency will be forced to grapple with many of these issues directly and deliberately in 2020as a result of deadlines of the agencys own making as well as external pressures coming from other parts of D.C. and from the rapidly changing nature of the U.S. health care system.

According to data presented by the Office of New Drugs in early December (see ONDs slides here), FDA had another extremely productive year when it comes to its approval of new molecular entities that address a unique blend of therapeutic areas. The agency approved 45 new molecular entities in FY 2019 (October1, 2018 to September30, 2019), of which 71%, or 32 products, received priority review status and 23 were designated as orphan drugs intended to treat rare diseases. As OND emphasized in the presentation, several of those new product approvals are notable for their uniqueness and therefore, in the agencys view, 2019 reflects not only quantity but [also] quality. The OND presentation also highlights a significant amount of other information on new molecular entity approvals and may be of interest to those readers who want to take a deeper dive into the data.

In addition to advancing important new drugs and biologics to market, former Commissioner Gottlieb is well-known for having spearheaded to development of a Drug Competition Action Plan (DCAP) and a Biosimilars Action Plan (BAP) during his nearly two-year tenure as head of the agency. Some of our prior coverage of the DCAP and BAP is available here. In general terms, the DCAP encourages market competition for generic drugs and helps to bring greater efficiency and transparency to the generic drug review process; the BAP aims to achieve similar goals for biosimilar products as the agency continues its implementation of the 2010 Biologics Price Competition and Innovation Act (BPCIA), including the critical drug-to-biologic transition that will occur by operation of law in March of 2020.

FDA continued to make progress on its various goals under these two initiatives during 2019. Some examples of this progress can be captured with these two data points:

FDA approved 1,171 generic drugs (935 full approvals and 236 tentative approvals) in the last fiscal year. Notably, 125 drug approvals were for first generics of medicines that had no direct generic competition. The agency released a report in October 2019 noting that generic drug approvals reached a record high in FY 2019 and also that several complex generic drug products had been approved for the first time. Under the DCAP, the agency has prioritized getting complex generic drug products to market with the goal of increasing competition and thereby reducing commercial prices for such products, which may be expensive due to difficult manufacturing processes.

On December 6, 2019, FDA licensed its 26th biosimilar product since the BPCIA was enacted and the 10th biosimilar this year (Amgens Avsola (infliximab-axxq), which is a biosimilar to Remicade and the fourth Remicade biosimilar approved by the agency to date). Although thirteen of the previous 25 approved biosimilars have yet to launch due to ongoing patent litigation or for business reasons, the agency has continued to focus on positive developments in the still-nascent U.S. biosimilars market, including with the issuance of a public statement on November 15 in conjunction with approval of the 25th biosimilar (see here).

With all of these wins, however, FDA still faces its fair share of challenges related to its expedited drug and biologic programs, especially as it appears to have accelerated its review of products intended for diseases with an unmet need to breakneck speed. Indeed, the OND presentation from early December also added that for FY 2020 and as of November 21, 2019, the agency had already approved 13 such drugs, suggesting that next year could be a record-breaking one in terms of innovative drug product approvals. A recent Bloomberg Law article (available here) used the phrase breakneck speed to describe the agencys actions in this space based on several recent FDA approvals of new molecular entities that have come months in advance of their assigned target dates. The Bloomberg Law article highlights that in response to FDAs speediness in reaching approval decisions on new drugs and biologics for diseases with unmet needs, patient advocates and, increasingly, insurers that have to pay for those treatments are starting to raise concerns that these products lack sufficient safety or effectiveness data.

Relatedly, there has been increasing pressure on FDA to remove certain accelerated approval drugs from the market following a failure by the drug product sponsor to confirm the efficacy or clinical benefits of the product in the required post-marketing confirmatory clinical trial. The most visible example of this regulatory challenge came in October 2019 when FDA convened an advisory committee to recommend whether it should withdraw accelerated approval from hydroxyprogesterone caproate injection (marketed under the brand name Makena for the prevention of preterm birth in pregnant women). FDAs Bone, Reproductive and Urologic Drugs Advisory Committee voted 9-7 to withdraw approval, with the dissenters favoring leaving Makena on the market while requiring the sponsor to conduct a new confirmatory trial. (Notably, no one voted for the option of leaving it on the market without requiring a new confirmatory trial.)Among the concerns of some members who voted to leave the product on the market with a new clinical trial obligation was that the drugs withdrawal would leave no safe treatment options for pregnant women at high risk of preterm birth. FDA will have to make a final decision regarding what to do about Makena in 2020, and it undoubtedly will face intense criticism (and potentially legal challenge) no matter what route it chooses to take for this public health quandary in which it finds itself.

In a similar vein, FDA official Dr. Richard Pazdur participated in a Senate briefing on December 10, 2019, in which he and other speakers defended the Breakthrough Therapy Designation program. FDA insisted again that the designation was intended to let the agency have earlier interactions with drug sponsorsand that it was not meant to be an early rating system for drugs or a signal of how they might do commercially. Given that the Breakthrough program was created in 2012 and is considered to be wildly successful, some speakers at the briefing expressed surprise that there was still any confusion about its purpose and function. Whether Congress picks up any of these emerging areas for consideration as part of FDAs 2022 user fee reauthorization packages remains to be seenas those negotiations will begin in earnest after the New Year, but the issues certainly are complex enough to allow for robust policy discussions to occur.

Finally, there are expected to be bumps in the road with the upcoming March 2020 transition of proteins previously approved under New Drug Applications (NDAs) to Biologics License Applications (BLAs)for which FDA only finalized its guidance for industry last year. The March 2020 transition date was established under the BPCIA and the agency does not have discretion in getting the transition done (only in how it handles the logistical and administrative issues created by transitioning approved products in this way). Check out our prior blog post on the final deemed to be a license transition guidance.

FDA guidance documents for all regulated product categories continued to be released on a regular basis this year, including several related to areas of agency priorities under the DCAP and BAP including the final biosimilar interchangeability guidance issued in May (see our blog post here) and a draft guidance on insulin interchangeability issued in November 2019. The latter also relates to the March 2020 NDA-to-BLA transition, as insulins are one of the largest class of products that will be transitioning into regulation as biologics, making them open to what is expected to be more efficient competition through the BPCIAs biosimilar pathway than what was possible in the past as insulin NDAs.

Despite the accelerated pace of the issuance of Agency guidance, however, FDA is beginning to face more challenges related to its decision-making and the scope of its exercise of agency discretion. In particular, a significant District Court for the District of Columbia ruling issued on December 6, 2019, Genus Medical Technologies, LLC v. FDA, provides hints of a potential shift in judicial deference to certain agency actions. The court vacated FDAs classification of a medical imaging liquid as a drug rather than as a device after determining that FDA did not have discretion to decide how to regulate a product merely because the definitions of drug and device overlap in the FD&C Act.

According to the district court judge, FDA was not interpreting the drug/device definitions in the statute properly, and Congress did not intend to allow the agency unfettered discretion to pick between the two categories. Rather, the court found that the text of the definitions are clear and do not create a gap or any ambiguity for FDA to fill with an exercise of agency discretion. This recently issued decision may indicate a potential shift in how courts are going to apply long-standing precedents related to judicial deference to agency decisions. If FDA decides to appeal the Genus ruling, it may end up at the Supreme Court as one of many expected challenges to the doctrines that established our current framework for judicial deference of an administrative agencys interpretation of an ambiguous statute.

One of the first FDA press releases for 2019 was co-authored by former Commissioner Gottlieb and Center for Biologics Evaluation and Research (CBER) Director Peter Marks and was focused on the agencys new policies aiming to advance the development of safe and effective cell and gene therapies. In the press release, the agency leaders predicted that by 2020, FDA would receive more than 200 Investigational New Drug Applications (INDs) for cell and gene therapies each year. The agency has continued to work diligently to increase its staff in CBER to conduct clinical reviews for such INDs and to try to keep pace with the industrys development of these innovative technologies.

As we discussed in our update on FDAs Comprehensive Regenerative Medicine Policy Framework earlier this year, the agency is prioritizing two parallel goals: (1) clarifying the regulatory criteria for product marketing and providing support and guidance to legitimate product developers; and (2) removing unapproved, unproven, and potentially unsafe products from the U.S. market. The second prong of this comprehensive plan for regenerative medicine products was the topic of one of Dr. Gottliebs very last statements as Commissioner before he left the agency, issued on April 3, 2019 in conjunction with CBER Director Dr. Marks, indicating how important this area is to the agencys current public health priorities.

In the April 2019 statement, Drs. Gottlieb and Marks acknowledged FDAs challenges and efforts to stop stem cell clinics and manufacturers from marketing unapproved products that put patients at risk, citing several Warning Letters issued to manufacturers that violated current good manufacturing practices (CGMPs) for human cells and tissue products. They noted that it was of particular concern given that the industry was nearly halfway through the period during which the FDA intends to exercise enforcement discretion for certain regenerative medicine products with respect to INDs and premarket approval requirements. Now that it is December 2019, that deadline is even closer with less than one year left. November 2020 is the end of the three-year period of enforcement discretion announced by FDA when it first articulated the policies and goals of this comprehensive framework in 2017. See our prior posts on the topic here and here.

Under the Comprehensive Regenerative Medicine Policy Framework, FDA appears to have stepped up the pace of issuing Warning and Untitled Letters to sellers of unapproved stem cell products during the second half of 2019. In conjunction with a Warning Letter issued on December 5, 2019 to two related companies for processing and marketing unapproved umbilical cord blood-derived cellular products, Dr. Marks of CBER reiterated the agencys concerns about safety and reminded the public of the upcoming compliance deadline: As evidenced by the number of actions that the agency has taken this month alone, there are still many companies that have failed to come into compliance with the [FD&C Act] and FDAs regulations.

Dr. Marks was referring to two Untitled Letters that were issued to stem cell product distributors on November 20 and November 25, 2019, respectively. The press release cited above also added that the agency had also recently sent 20 letters to manufacturers and health care providers noting that it has come to [FDAs] attention that they may be offering unapproved stem cell products, reiterating the FDAs compliance and enforcement policy.

FDA also prevailed this year in the U.S. District Court of the Southern District of Florida against a stem cell clinic charged with violating the FD&C Act and the PHS Act. In June 2019, the court held that the defendants adulterated and misbranded a stem cell drug product made from a patients adipose tissue without FDA approval and for significant deviations from CGMPs, issuing a permanent injunction as requested by the Department of Justice on FDAs behalf. The agencys statement on that important court win by the government is available here.

Lastly, on December 6, 2019, FDA issued a Public Safety Notification on Exosome Products. The safety notification informed the public of multiple recent reports of serious adverse events experienced by patients in Nebraska who were treated with unapproved products marketed as containing exosomes, which came to FDAs attention through the Centers for Disease Control and Prevention, the Nebraska Department of Health and Human Services, and others. There are currently no FDA-approved exosome products and, to be honest, we are not even sure what such a product would be since an exome consists of all the sequenced exons within a single human genome after the introns are removed. (So were the clinics administering complete exomes to patients? that seems unlikely.)But what we found noteworthy about this public safety notice is the forceful and direct language FDA used when describing the unscrupulous conduct of the sellers of these products:

Certain clinics across the country, including some that manufacture or market violative stem cell products, are now also offering exosome products to patients. They deceive patients with unsubstantiated claims about the potential for these products to prevent, treat or cure various diseases or conditions. They may claim that they these products do not fall under the regulatory provisions for drugs and biological products that is simply untrue. As a general matter, exosomes used to treat diseases and conditions in humans are regulated as drugs and biological products under the [PHS Act] and the [FD&C Act] and are subject to premarket review and approval requirements.

The clinics currently offering these products outside of FDAs review process are taking advantage of patients and flouting federal statutes and FDA regulations. This ultimately puts at risk the very patients that these clinics claim to want to help, by either delaying treatment with legitimate and scientifically sound treatment options, or worse, posing harm to patients, as evidenced by these recent reports of adverse events.

As we enter the final year of FDAs enforcement discretion period, perhaps these public notices and Warning/Untitled Letters will become even more frequent and the agency will become even more frustrated by the ongoing violations and medical practitioners who flout federal law. This area will see substantial activity in 2020 and we will be watching closely to see what changes, if anything, about FDAs approach in November when the deadline to come into compliance ends. Will there be widespread FBI raids on stem cell clinics engaged in this kind of bad behavior? Only time will tell.

Although we have highlighted what we view as some important challenges for the FDA to address in the coming months, other areas continue to be business as usual without anything very new to report. FDA continues to invest significant resources into improving the quality of compounded drugs and ensuring compliance with Sections 503A and 503B of the FD&C Act. Drug compounding was another topic of one of Dr. Gottliebs very last statements as Commissioner on April 3, 2019, in which he laid out the 2019 compounding priorities that included maintaining quality manufacturing and compliance and regulating compounding from bulk drug substances.

Notwithstanding all the efforts by FDA and State regulators in this area over the past several years, the agency continues to see concerning activity when it comes to compounded drugs, such as problems related to the condition under which compounded sterile medicines are made, which raisesignificant risks to patients. As a result, FDA has made it an intense area of focus to take enforcement actions against compounders who fail to produce sterile drugs in compliance with the law. During the past year, for example, FDA has won at least four permanent injunctions against various compounders after the agency identified behavior that posed a significant risk to public health and safety.

In addition, in 2019 FDA also increased its activities towards reducing and mitigating the impact of drug shortages on the health care system. See our prior blog post on Drug Shortages.

Finally, after a fairly slow year of enforcement in the prescription drug advertising space, the last two months of 2019, at least as of December 12th, have given us three (!) letters two untitled and one warning from the FDAs Office of Prescription Drug Promotion (OPDP). Most interestingly, the Warning Letter issued on December 2, 2019 for omitting warnings about the most serious risks associated with [a medication-assisted treatment] drug from promotional materials was announced to the public via FDA press release, which is not a typical action for normal-course OPDP letters to industry. The drug in question, approved for the prevention of relapse to opioid dependence following opioid detoxification, is associated with several significant risks including potential opioid overdose. Given the countrys public health emergency that is the opioid epidemic, FDA appears to have felt the need to make the deficiencies in the advertisement and those risks more widely publicized. So another thing we will be watching for in the New Year is whether this OPDP action represents the beginning of a new trend by the agency to publicize these Warning Letters more directly, or whether its advertising enforcement activities may be picking up due to industrys evolving approaches to promoting therapeutic products.

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Drugs, Biologics, and Regenerative Medicine in 2019 - The National Law Review