Duchenne muscular dystrophy
Sarepta nevertheless plans to push for full FDA approval of Vyondys 53 and Amondys 45 based on what it said are “encouraging trends” in efficacy.
The takeover of its competitor, announced Sunday, could also bring some attention to Dyne Therapeutics, which has a similar RNA-based pipeline in rare muscle diseases.
A new analyst survey suggests that doctors are still prescribing Sarepta’s Elevidys, even after a series of deaths in certain populations marred the gene therapy’s record.
Analysts at BMO Capital Markets expect the lack of other exon-44-skiping therapies to facilitate a “smooth” approval process for Avidity’s del-zota.
As AAV9 and CRISPR programs navigate safety, delivery and scalability hurdles, small molecules offer a deployable, scalable bridge, complementing genetic approaches and accelerating meaningful impact for patients with Duchenne muscular dystrophy.
Capricor Therapeutics met with the FDA last week for a type A meeting, during which CEO Linda Marbán aimed to explain to the regulator that it got it wrong. Capricor plans to resubmit the application based on deramiocel’s existing dataset.
Prasad Returns, Delany Departs, Lilly’s Weight Loss Pill Disappoints and Sarepta’s Fallout Continues
CBER Chief Vinay Prasad reclaimed his job less than two weeks after his mysterious exit; MAHA implementor Gray Delany is out after reportedly sparring with other agency officials over communications strategy; Eli Lilly’s first Phase III readout for oral obesity drug orforglipron missed analyst expectations; and Arrowhead Pharmaceuticals addresses the recent woes of its of partner Sarepta.
Jefferies analysts said these detailed safety outcomes confirm the gene therapy’s positive risk/benefit profile in ambulatory patients with Duchenne muscular dystrophy.
Sarepta did not hold an investor call for its second-quarter earnings report or provide an updated full-year revenue outlook.
George Tidmarsh takes over temporarily at CBER following Vinay Prasad’s abrupt departure; Replimmune trial leaders protest rejection reportedly driven by FDA’s top cancer regulator Richard Pazdur; Merck’s $3 billion savings push claims 6,000 jobs; and Pfizer CEO Albert Bourla addresses President Donald Trump’s new threats around Most Favored Nation drug pricing.
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