Roche halts clinic studies of delandistrogene moxeparvovec for people who are non-ambulatory
Roche has announced that it is halting treatment with gene therapy drug delandistrogene moxeparvovec (also known as ELEVIDYS, or dela mox, and SRP-9001) in people with Duchenne muscular dystrophy (DMD) who are non-ambulant (not walking), and regardless of their age.
It has said that this decision does not impact the treatment of people with DMD who are ambulatory (walking) of any age, where the benefit-risk ratio remains positive and unchanged.
Halt of clinical studies
It follows the news that two young people who were treated with it passed away due to liver failure.
European regulators requested that Roche and Sarepta (the company that is producing it in the US) temporarily halt clinical studies 104 (NCT06241950), 302 (ENVOL, NCT06128564) and 303 (ENVISION Study 303, NCT05310071) after that. The clinical studies remain halted.
Roche has said that dosing for the ENVISION study outside Europe will be paused immediately.
Gene therapy
Delandistrogene moxeparvovec 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.
Future of delandistrogene moxeparvovec
Roche says that it is committed to ongoing monitoring and evaluation of the benefit-risk profile of delandistrogene moxeparvovec for people with DMD.
A health technology appraisal by the National Institute for Health and Care Excellence (NICE) on delandistrogene moxeparvovec for treating DMD in children aged four to seven years is scheduled for the autumn.