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Sarepta must also run a post-marketing study for Elevidys to better assess the risk of serious liver injury in patients dosed with the gene therapy.

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.

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.

Despite announcing a broad pivot to siRNA earlier this year, Sarepta is following through with an investigational gene therapy: its limb-girdle muscular dystrophy candidate. But the treatment’s path forward, analysts say, is highly uncertain.

After the FDA revoked Sarepta Therapeutics’ platform designation in July, Krystal Biotech is “poised to be the first major beneficiary” of this program, according to analysts at William Blair.

CBER is unanimously against Elevdiys’ return to the market without additional evidence, according to media reports citing an anonymous senior FDA official. Given Elevidys’ full approval, however, experts told BioSpace this path would set up a length legal battle between the regulator and Sarepta Therapeutics.

Pfizer and Novo Nordisk continue to fight for ownership of obesity startup Metsera; CDER Director George Tidmarsh leaves his position amid an ongoing probe into his “personal conduct”; FDA reverses course on approval requirements for uniQure’s Huntington’s gene therapy; Sarepta’s exon-skipping Duchenne muscular dystrophy drugs fail confirmatory study.

In the wake of multiple patient deaths from liver injuries related to Sarepta Therapeutics’ AAV gene therapy platform, some in the sector are looking for ways to improve the current technology, while others are eager to move on.

Sarepta Therapeutics’ stock has dropped precipitously as questions swirl around the safety of its gene therapies. Meanwhile, the Duchenne patient community fears losing access to Elevidys while the regulator considers more drastic action.