- Sarepta said the Food and Drug Administration had requested more data on its drug for Duchenne muscular dystrophy (DMD), causing shares of the company to jump in late trading Monday evening.
- The FDA had originally been expected to decide on final approval for the drug, called eteplirsen, by May 26, but said late last month it would not meet its deadline.
- Sarepta will submit data from 13 patient biopsy samples from an ongoing study of eteplirsen to help support its application.
The FDA's decision last month to extend its review of eterplirsen had raised hopes for some sort of approval, or at least a quicker path to resubmission. But an announcement by the regulator one week later making it easier for companies to sell unapproved drugs at manufacturing cost through limited compassionate use programs brought those hopes crashing back down.
Some had seen the announcement as a way for the FDA to reject the drug while still allowing patients to obtain it. However, Janet Woodcock, head of the FDA's Center for Drug Evaluation and Research, has said there is no connection between the two, according to the Boston Business Journal.
Now shares of Sarepta have climbed again following news of the FDA's request, highlighting eteplirsen's precarious position and the hyper-attention to the drug's fate.This focus has only increased after BioMarin, another company in the space, announced it would halt current development of DMD drugs and withdraw an application for European approval of its main drug drisapersen.
The FDA's rejection of drisapersen and another DMD drug from PTC Therapeutics earlier this year has left eteplirsen as the DMD patient community's sole near-term hope for a treatment. Patients, doctors, and advocates have pushed for the FDA to approve eteplirsen based on a small trial of 12 boys which seemed to show dramatic benefit in slowing symptoms.
But FDA staff and an independent advisory panel have criticized the trial's design and results, leaving analysts expecting a rejection for the drug.
The request for more information adds another twist to the process. Sarepta will submit the data over the coming weeks to "facilitate a prompt decision" by the FDA.