Sarepta Therapeutics’ Gene Therapy Gets Major FDA Approval – Finimize

Whats going on here?

Sarepta Therapeutics' shares surged around 36% in premarket trading after the FDA expanded its approval of the gene therapy Elevidys.

What does this mean?

Sarepta received full FDA approval for Elevidys for ambulatory patients aged four and above and accelerated approval for those who cannot walk. This move significantly boosts the therapys market reach, with projections anticipating sales to hit $3 billion by 2025 and peak at $5 billion by 2027. In 2023, Elevidys generated $200.4 million in revenue, with expectations set to soar to $991.91 million this year. Given that Duchenne Muscular Dystrophy (DMD) affects roughly one in 3,500 male births worldwide, Elevidys' expanded approval now targets about 13,000 US patients covering nearly 90% of the US DMD market.

Why should I care?

For markets: The gene therapy gold rush.

In the evolving landscape of gene therapies for DMD, Sarepta's Elevidys is positioned to be the dominant player with no substantial competition until post-2027. RegenxBio is exploring early to mid-stage trials for a competing therapy, while Pfizer faced a setback with a recent late-stage trial failure. This dominance translates into significant market stability for Sarepta, backed by robust financial forecasts.

The bigger picture: The horizon looks bright.

With the global DMD treatment market projected to grow to $11.47 billion by 2034, the expanded use of Elevidys places Sarepta in a commanding position. The company's investor call scheduled for 08:30 am ET is expected to shed light on strategic plans moving forward. At $3.2 million per treatment, Elevidys might be one of the priciest therapies globally, but its value proposition and market penetration could redefine the treatment landscape for DMD.

Originally posted here:
Sarepta Therapeutics' Gene Therapy Gets Major FDA Approval - Finimize