By Zachary Stieber
Contributing Writer
In a reversal, the drugmaker Sarepta Therapeutics said July 21 it is stopping all shipments of a gene therapy that has been linked to multiple deaths.
Sarepta said in a statement it would voluntarily, and temporarily, halt all shipments of Elevidys at the close of business on July 22.
Elevidys is a gene therapy treatment for Duchenne muscular dystrophy, a disorder that is inherited and features weakening muscles and increasing difficulty moving.
There is no cure, but treatments and physical therapy can help control the condition.
Two non-ambulatory patients died after receiving Elevidys. The first death was reported in March and the second was reported by Sarepta in June. Sarepta then stopped shipments of Elevidys to non-ambulatory patients as it worked on a solution.
A third patient, also non-ambulatory, died after receiving a similar therapy for a different type of muscular dystrophy, Sarepta said on July 19.
The Food and Drug Administration requested Sarepta pause all shipments of Elevidys, but Sarepta initially refused. The drugmaker said on July 18 that its analysis of the data “shows no new or changed safety signals in the ambulant patient population,” so “we will continue to ship Elevidys to the ambulant population.”
In the update on Monday, Sarepta said halting all shipments would enable it to respond to requests for information, and work with regulators to complete a supplement to labeling for the drug.
“As a patient-centric organization, the decision to voluntarily and temporarily pause shipments of Elevidys was a painful one, as individuals with Duchenne are losing muscle daily and in need of disease-modifying options,” Douglas Ingram, Sarepta’s CEO, said in a statement.
“It is important for the patients we serve that Sarepta maintains a productive and positive working relationship with FDA, and it became obvious that maintaining that productive working relationship required this temporary suspension while we address any questions that FDA may have and complete the Elevidys label supplement process.”
The FDA did not respond to a request for comment.
The third death was in a patient who received an unapproved gene therapy for limb-girdle muscular dystrophy. The gene therapy utilizes the same serotype as Elevidys.
The agency said in a statement on July 18 that it was placing clinical trials for Sarepta’s investigational gene therapy for limb-girdle muscular dystrophy on hold following the deaths, which it described as “potentially related to these products.”
The FDA has also revoked Sarepta’s platform technology designation.
