The third quarter marked a momentous one for Sarepta Therapeutics, bringing the long-anticipated launch of its Duchenne muscular dystrophy (DMD) gene therapy Elevidys.
After traveling a tumultuous road to the June approval, Sarepta reaped $69.1 million from Elevidys' first quarter on the market. The number was more than enough to beat analysts' expectations of between $20 million and $24 million.
Sarepta stayed mum on exactly how many patients have received an infusion and instead provided revenue as a marker for uptake, CEO Doug Ingram said on Sarepta’s third-quarter earnings call. The drug is priced at $3.2 million per infusion.
Also on the call, Sarepta reiterated its confidence that its phase 3 EMBARK study can potentially support a label expansion for Elevidys despite missing its primary endpoint.
The company's discussions with FDA leadership have shown that the agency is open to the idea of a label expansion “if supported by review of the data," Sarepta said in its earnings release. The company plans to submit a supplemental application “very soon,” Ingram added.
For Elevidys' initial accelerated approval, Sarepta's application went through an FDA advisory committee, which ultimately voted in favor of the therapy. This time around, Ingram feels “pretty confident” that the label expansion wouldn't warrant an advisory committee discussion, a standpoint he says is shared by FDA leadership.
For a potential label expansion, Sarepta is seeking FDA approval to treat all DMD patients. Currently, the drug's label covers ambulatory patients ages 4 to 5.
While Ingram held firm that the EMBARK study showed evidence of benefit from the gene therapy, the trial results demonstrated that Elevidys wasn’t able to meet statistical significance in its primary endpoint measuring motor response on the North Star Ambulatory Assessment scale.
Despite the trial miss, Ingram says parents of patients outside of the currently approved age range are “probably even more compelled” to want to start the therapy and are awaiting the expanded label.
All in all, Sarepta generated third-quarter revenues of $309.3 million, representing 49% growth from the same period in 2022. The company reaffirmed its financial guidance of more than $925 million in product revenue for the full year.

https://www.fiercepharma.com/pharma/sarepta-enjoys-early-sales-ramp-dmd-gene-therapy-maintains-confidence-label-expansion