The U.S. Food and Drug Administration (FDA) has decided to move forward with a meeting of its advisory committee regarding Sarepta’s investigational gene therapy for Duchenne muscular dystrophy (DMD) before its original May 29 action date. This meeting is a pivotal step in making the groundbreaking therapy available to those diagnosed with DMD.
Sarepta President and CEO Doug Ingram has announced that the regulator has found no major safety issues with SRP-9001 (delandistrogene moxeparvovec), and as a result, has no intention of holding an advisory committee meeting.
Shares of Sarepta dropped sharply Thursday evening, with a 20% slide in after-hours trading, after news broke that there is still no set date for the company’s adcomm. Investors were clearly disappointed by the news.
Ingram reassured investors on Thursday that the safety of the candidate for the 9001 construct is not a major concern, and the primary focus of the meeting will be the design of the 9001 construct and the evidence that 9001 dystrophin is likely to bring about clinical benefit.
No new evidence or data was needed to prompt the adcomm’s decision regarding delandistrogene moxeparvovec, according to Ingram. Sarepta does not anticipate any major delays in their target action date, nor does it anticipate the need to file a major amendment to the candidate’s Biologics License Application (BLA).
Sarepta’s Chief Executive Officer, Doug Ingram, expressed optimism in the face of the FDA’s decision to convene an adcomm meeting to discuss delandistrogene moxeparvovec, stating that the company had always expected such a panel and had prepared accordingly.
DMD is a devastating neuromuscular disorder affecting children and adults worldwide. Caused by mutations in the dystrophin gene, this condition manifests as developmental delays and muscle weakness. But hope is on the horizon – Delandistrogene moxeparvovec is an innovative gene therapy designed to restore the dystrophin gene and improve the lives of those suffering from DMD.
The FDA granted priority review to Sarepta’s Biologics License Application (BLA) for their innovative drug, delandistrogene moxeparvovec, in November 2022, allowing for its potential approval and distribution. This approval could greatly expand treatments available to those with certain rare diseases.
Over 80 patients enrolled in three studies – SRP-9001-101, SRP-9001-102 and SRP-9001-103 – were part of a comprehensive body of evidence that showed the efficacy of a therapy over different time points. The results of these studies formed the basis of the BLA.
Initial studies of Sarepta’s candidate drug, delandistrogene moxeparvovec, showed promise in increasing dystrophin expression. However, the effects on functional endpoints were initially a bit mixed. Recent results, however, demonstrate a marked improvement in functional outcomes, giving hope for a much-needed treatment for this condition.
During a late-cycle meeting, the FDA informed Sarepta that no significant safety issues with the gene therapy had been identified and that no risk management program would be necessary. According to Sarepta’s CEO Ed Ingram, the FDA did not anticipate the need for any such program.