Roche temporarily halts clinic studies
Following the terrible news that a young man who was treated with Duchenne muscular dystrophy (DMD) gene therapy drug ELEVIDYS tragically passed away, Sarepta and Roche have temporarily halted several of their clinical studies. Roche, the company that the treatment is licenced to outside the US, has said that this was at the request of the European Medicines Agency (EMA).
Clinical studies halted
The pharmaceutical companies have put a temporary clinical hold on Study 104 (NCT06241950), Study 302 (ENVOL, NCT06128564), and Study 303 (ENVISION, NCT05881408) in the EU until the analysis into the cause of death is complete.
Roche is also pausing enrolment and dosing of participants in the UK for its ENVOL study. Patient safety monitoring for already enrolled participants and ongoing collection of data will continue.
Roche has issued a letter to the community which you can read here.
ELEVIDYS
ELEVIDYS (also known as delandistrogene moxeparvovec, or dela mox, and SRP-9001) is an adeno-associated virus (AAV) vector-based gene therapy product for ambulatory patients with a confirmed mutation in the DMD gene. It is a one-time treatment designed to treat the underlying cause of DMD.
Sarepta, the company that is producing it in the US, has said that the young man died from acute liver failure. It said that acute liver injury is a known possible side effect of ELEVIDYS and other AAV-mediated gene therapies.
Sarepta said that, although it is not a new safety signal, acute liver failure (ALF) leading to death represents a severity of acute liver injury not previously reported for ELEVIDYS, which to date has been used to treat more than 800 patients in clinical trials or as a prescribed therapy.
Sarepta said that testing revealed that the young man had a recent cytomegalovirus (CMV) infection which was identified by the treating physician as a possible contributing factor. CMV can infect and damage the liver, a condition known as CMV hepatitis.
ELEVIDYS in the UK
Although Roche’s trials for ELEVIDYS have been halted, Sarepeta’s outside the European Union are continuing – including Study 104 and ENVISION within the UK.
If you have questions about these trials, we recommend that you speak to your neuromuscular consultant about it.