Three ongoing clinical trials of ELEVIDYS (delandistrogene moxeparvovec-rokl), a gene therapy developed by Sarepta and marketed by Roche, have been temporarily halted by the European Medicines Agency (EMA). This expected but not unwelcome news has caused deep sadness in the Duchenne muscular dystrophy (DMD) community.
The paused trials include the Sarepta-sponsored phase 3 Envision study, which is evaluating Elevidys in boys aged 8 to 17; Roche’s phase 2 Envol study, which is investigating Elevidys in babies and newborns; and Sarepta’s Study 104, a phase 1 look at Elevidys in boys ages 4 to 9 with pre-existing antibodies to a particular serotype.
Before Roche, Sarepta and EMA the first community to publish this announcement was our Turkish partner >>> DMD Dayanisma.
Roche has issued a letter to the community which you can read here. (PDF)
Table of Contents
Why Were Elevidys Clinical Trials Halted?
After the death of a 16-year-old boy with Duchenne muscular dystrophy who received an infusion of Elevidys on March 18, 2025, the outlook for the gene therapy, which is marketed at a commercial price of $3 million, changed abruptly. (Read More)
Until the precise cause of death of a U.S. teen who developed acute liver failure (ALF) after taking Elevidys can be ascertained, the European Medicines Agency (EMA) requested that the studies be put on hold. When the patient passed away last month, Sarepta declared that it will amend Elevidys’ label to include the safety signal.
SRP-9001-104 Temporarily Halted
Source: Clinical Trial of SRP-9001-104
ENVOL (Study 302) Temporarily Halted
Source: Clinical Trial of SRP-9001-302
SRP-9001-303 Temporarily Halted
Source: Clinical Trial of SRP-9001-303
Learn More: DMD WarrioR’s Turkish Representative Shares His Views on Elevidys Gene Therapy.
