The FDA won’t be holding an advisory panel meeting for Sarepta Therapeutics’ SRP-9001, which is now that much closer to becoming the first gene therapy for Duchenne muscular dystrophy (DMD).
“2023 may be the most event eventful year in Sarepta’s event-filled history,” President and CEO Doug Ingram said on a Feb. 28 earnings call held after market close.
In November, the FDA accepted Sarepta’s biologics license application seeking accelerated approval for SRP-9001. The agency has now set May 29 as the expected decision date. A midcycle review related to the process has already occurred, Ingram said, and the FDA did not flag any significant safety issues associated with SRP-9001.
The gene therapy is designed to deliver the microdystrophin-encoding gene into muscle tissue to prompt production of the microdystrophin protein. Patients with DMD have a mutation in the DMD gene and can’t make the protein on their own, leading to a progressive loss of muscle strength.
During the midcycle review, the FDA said it would not require an advisory committee, which is used to provide the agency with independent recommendations for drug products about to enter the market.
Beyond that, Ingram said he doesn’t have any additional color to provide as questions started pouring in on the earnings Q&A about the regulatory activity.
“We had the midcycle,” the CEO explained. “They confirmed they saw no significant safety issues with the program to be able to identify any significant clinical issues or major deficiencies and then determined that they didn’t need an AdComm. That’s where we are right now.”
Sarepta has moved fast to prepare the resources to bankroll the therapy’s launch once the FDA gives its OK, raising $1.2 billion in preparation. In January, CDMO giant Catalent inked a deal with Sarepta to manufacture SRP-9001, with all manufacturing inspections scheduled, according to Ingram.
As of Dec. 31, the Cambridge, Massachusetts-based company recorded net revenue of $843.8 million for 2022, up from $612.4 million the year prior.
Sarepta’s stock has surged 20% since closing yesterday at $122.13 per share, rising to $146 as of 9:30 a.m. ET today.