Press Release: Rilzabrutinib granted orphan drug designation in the US for two rare diseases with no approved medicines

Posted: April 3, 2025 at 2:45 am

Rilzabrutinib granted orphan drug designation in the US for two rare diseases with no approved medicines

Here is the original post:
Press Release: Rilzabrutinib granted orphan drug designation in the US for two rare diseases with no approved medicines

Related Posts

Eton Pharmaceuticals Announces Closing of Public Offering [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
Mydecine Innovations Group Appoints Boustead Capital Markets LLP as Financial Advisor for its Planned Dual Listing on the London Stock Exchange [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
Agios Announces Withdrawal of European Marketing Authorization Application for TIBSOVO® as a Treatment for Relapsed or Refractory IDH1-mutant Acute... [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
Stabilization measures taken [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
Coherus BioSciences Announces New Employment Inducement Grants [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
Biomerica Reports Fiscal 2021 1st Quarter Financial Results [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
German Federal Joint Committee (G-BA) Issues Nationwide Reimbursement Decision for EndoPredict® Breast Cancer Prognostic Test [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
Y-mAbs Announces Update on Naxitamab and Omburtamab in Neuroblastoma [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
Psychedelics Experiencing Renaissance, Offer Tremendous Treatment Potential [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
SPACs Show Investors Faster, Simpler Way to Go Public [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
Spero Therapeutics to Present Data for All Pipeline Programs at IDWeek 2020 [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
Zogenix Receives Positive CHMP Opinion for FINTEPLA® (Fenfluramine) Oral Solution for the Treatment of Seizures in Patients with Dravet Syndrome [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
CHMP recommends approval of Dupixent® (dupilumab) for children aged 6 to 11 years with severe atopic dermatitis [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
Beyond Air® Presents Positive New Preclinical Data for the Use of a Single Injection of Gaseous Nitric Oxide as a Novel In situ Cancer Vaccination [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
Aimmune Receives Positive CHMP Opinion on PALFORZIA® for the Treatment of Patients with Peanut Allergy in Europe [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
Novartis receives positive CHMP opinion for Leqvio®* (inclisiran), a potential first-in-class siRNA for the treatment of high cholesterol [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
Nephros Announces Pricing of $5 Million Registered Direct Offering of Common Stock [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
Theratechnologies Appoints Two New Board Members [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
Cantor Fitzgerald and Kristen Kluska to Host Zoom Fireside Chat with Ocugen [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
First Patient Dosed in Phase II Trial to Treat Complications in COVID-19 [Last Updated On: October 17th, 2020] [Originally Added On: October 17th, 2020]
Basilea announces Clinical Trial Collaboration and Supply Agreement with Eli Lilly and Company for ramucirumab in the ongoing FIDES-03 study with... [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
Novartis announces collaboration with Molecular Partners to develop two DARPin® therapies designed for potential use against COVID-19 [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
Supernus to Host Third Quarter 2020 Financial Results Conference Call [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
CLINUVEL to Trial Innovative Drug in Stroke [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
Sanofi and GSK to support COVAX with 200 million doses of adjuvanted, recombinant protein-based COVID-19 vaccine [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
Repligen Corporation Announces Agreement to Acquire Bioprocess Systems Innovator ARTeSYN Biosolutions and Completes Acquisition of Non-Metallic... [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
Transactions in connection with and conclusion of share buyback program [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
Vir Biotechnology to Provide Corporate Update and Report Third Quarter 2020 Financial Results on November 10, 2020 [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
Opiant Pharmaceuticals to Report Third Quarter 2020 Financial Results and Host Conference Call and Webcast on November 12, 2020 [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
Aptose to Release Third Quarter Ended September 30, 2020 Financial Results and Hold Conference Call on November 10, 2020 [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
Lumos Pharma to Report Third Quarter 2020 Financial Results and Host a Conference Call on November 10, 2020 [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
Galera Therapeutics Announces Interim Data from Pilot Phase 1/2 Trial of GC4419 in Combination with Stereotactic Body Radiation Therapy Showed... [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
Sanofi: Information concerning the total number of voting rights and shares - September 2020 [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
Indus Holdings, Inc. to Report Third Quarter 2020 Financial and Operational Results [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
Galapagos’ R&D Roundtable showcases Toledo program [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
Tiziana Life Sciences Announces Timetable of Demerger of Accustem (for holders of Tiziana ADRs) [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
Novavax Provides Phase 3 COVID-19 Vaccine Clinical Development Update [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
CohBar to Participate at ROTH Capital COVID-19 Therapeutics in Development Event [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
Akari Therapeutics to Present Phase II Data from Bullous Pemphigoid Trial at European Academy of Dermatology and Venereology (EADV) Virtual Congress... [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
BioStem Technologies, Inc. Announces Launch of AEON™ [Last Updated On: October 28th, 2020] [Originally Added On: October 28th, 2020]
electroCore to Present at the 13th Annual LD Micro Main Event Conference [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Auris Medical Holding to Present at the 13th Annual LD Micro Main Event Conference [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Agios Announces Updated Data from Phase 1 Study of Mitapivat, First-in-Class PKR Activator, in Sickle Cell Disease [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Avid Bioservices Announces Appointment of Jeanne Thoma to Board of Directors [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Tricida to Provide an End-of-Year Business Update [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Annovis Bio to Present at Benzinga Global Small Cap Conference on December 8, 2020 [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Trillium Therapeutics Presented Clinical Data at the 62nd ASH Annual Meeting and Provides Guidance for 2021 [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Harpoon Therapeutics to Host Clinical Pipeline Programs Update Call and Webcast on December 8, 2020 [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Inventiva announces the publication in the Journal of Hepatology of new pre-clinical data showing the beneficial effects of lanifibranor on cirrhosis [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Opiant Pharmaceuticals Announces Appointment of Biopharmaceutical Leader Craig A. Collard as Chairman to its Board of Directors [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Signal Relief Patch Reviews – Pain Patch launched - Product Review by Mike Vaughn [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Autolus Therapeutics presents additional data on AUTO3 in DLBCL during the 62nd ASH Annual Meeting [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Cellworks CBM Biosimulation Identifies Genomic Causes for Induction Failure in AML Patients and Suggests Alternative Therapies [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Cellworks Personalized Biosimulation Clinical Trials Achieve 90% Therapy Response Prediction Accuracy for AML and MDS Patients [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Cellworks Personalized Biosimulation Clinical Trial Predicted ATO and ATRA Therapy Response in APL Patients with 93% Accuracy [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Cellworks CBM Identifies Genomic Signatures Impacting HOXA Regulation that Determine Response for AML Patients with Monosomy 7 [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
TG Therapeutics Announces Pivotal Data from the UNITY-CLL and UNITY-NHL Clinical Trials Presented at the 62nd American Society of Hematology Annual... [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Nurix Therapeutics Presents Preclinical Data at 62nd American Society of Hematology (ASH) Annual Meeting and Exposition [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Affimed Announces Updated AFM13 Clinical Data Presentation at the American Society of Hematology Annual Meeting, Demonstrating Good Tolerability and a... [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Kymera Therapeutics Presents Preclinical Data on IRAKIMiD and STAT3 Programs at Virtual 62nd American Society of Hematology (ASH) Annual Meeting [Last Updated On: December 7th, 2020] [Originally Added On: December 7th, 2020]
Avicanna Expands Distribution Network in South America Through Commercial Partnerships in Ecuador and Commercial Export of Feminized Seeds to Uruguay [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
Kolab Project Launches 232 Series Limited Edition Live Terpene 510 Vape Cartridges [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
Progenity Expands Availability of COVID-19 PCR Testing Services Across United States [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
NextCure Provides Updates on NC318 Clinical Program [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
C4 Therapeutics to be Added to the Russell 2000® and Russell 3000® Indexes [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
BioXcel Therapeutics to Present at the 39th Annual J.P. Morgan Healthcare Conference [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
miRagen Enters into License Agreement with Xencor for use of Xtend™ Antibody Technology for Investigational Antibodies to Treat Thyroid Eye Disease [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
Sigilon Therapeutics Receives Orphan Drug Designation for SIG-005 for the Treatment of Mucopolysaccharidosis Type I [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
Otonomy Reports Positive Top-Line Results from Phase 1/2 Clinical Trial of OTO-413 in Patients with Hearing Loss [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
Avenue Therapeutics Provides Regulatory Update for IV Tramadol [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
QuantuMDx embarks on £11 million scale-up to mass manufacture its diagnostic Q-POCTM device and disposable test cassette [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
ALX Oncology Added to the NASDAQ Biotechnology Index [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
Surface Oncology Announces Exclusive License Agreement with GSK for Novel Immunotherapy Program [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
Annovis Bio Interview to Air on Bloomberg International on the RedChip Money Report [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
Zynerba Pharmaceuticals Provides Regulatory Update on Zygel™ in Fragile X Syndrome [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
Publication of Supplementary Prospectus [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
Global WholeHealth Partners Corp (OTC: GWHP) Has Paid Off One of Its Outstanding Convertible Notes [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
Glycotope announces licensing agreement with ONK Therapeutics for humanized GlycoBody targeting TA-MUC1 [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
Transactions in Zealand Pharma shares and/or related securities by persons discharging managerial responsibilities and/or their closely associated... [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]
Arecor's Partner Inhibrx Exercises Its Option to License a New Formulation Developed by Arecor in Accordance With the Terms of Formulation Development... [Last Updated On: December 17th, 2020] [Originally Added On: December 17th, 2020]