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An OIG report zeroed in on what it said were three particularly problematic accelerated approvals: Biogen’s Aduhelm, Sarepta’s Exondys and Covis’ Makena.

Sarepta Therapeutics’ Duchenne muscular dystrophy therapy Elevidys handily beat analysts’ expectations in the fourth quarter of 2024, reflecting the biotech’s “world-class” execution, according to BMO Capital Markets analysts.

With Sarepta’s gene therapy Elevidys now available to a majority of Duchenne muscular dystrophy patients, experts express cautious optimism while emphasizing the need for further data.

Not exactly known for its dealmaking, Sarepta Therapeutics has thrown down a massive wad of cash to work with Arrowhead Pharmaceuticals on RNAi-based medicines.

Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for 2026—potentially posing a threat to Sarepta’s Elevidys.

The investigational therapy, vesleteplirsen, had been positioned as an updated version of Sarepta’s original exon 51-skipping Duchenne muscular dystrophy drug Exondys 51.

With Elevidys expansion in hand, Sarepta commits up to $10 billion to develop short interfering RNA–based drugs to build out its pipeline.

Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, granted equity awards on June 28, 2024 that were previously approved by the Compensation Committee of its Board of Directors under Sarepta’s 2024 Employment Commencement Incentive Plan, as a material inducement to employment to 23 individuals hired by Sarepta in June 2024.