Sarepta says will continue to ship Elevidys to ambulant population

Sarepta (SRPT) Therapeutics issued a statement which reads in part: “Shortly after 2:30 p.m. ET today, Sarepta received an informal request from the U.S. Food and Drug Administration to voluntarily halt shipment of Elevidys, our gene therapy for Duchenne muscular dystrophy, in the U.S. We first heard of this potential request earlier in the day at the same time the public and our patient communities did, through media reports…Based on our comprehensive scientific interpretation of the data, which shows no new or changed safety signals in the ambulant patient population, we will continue to ship Elevidys to the ambulant population. We look forward to continued discussions and sharing of information with FDA in order to advance our shared purpose of protecting patient safety and informed access to care. We recognize that the death of any patient is heartbreaking, including the recent death of a 51-year-old non-ambulant Limb-Girdle Muscular Dystrophy (LGMD) patient. We also want to clarify that this tragic event occurred in a Phase 1 clinical trial for an investigational gene therapy called SRP-9004. SRP-9004 is a clinical stage therapy that is intended to treat a different disease (LGMD Type 2D), is administered using a different dose, and is manufactured using a different process. The LGMD study participant who passed away was not treated with ELEVIDYS, and the dosing for the SRP-9004 trial had concluded at the time of his death.”