FDA in US removes voluntary hold of ELEVIDYS for ambulatory patients
The Food and Drug Administration (FDA) in the US has announced that it is now recommending the removal of the voluntary hold of ELEVIDYS for ambulatory (walking) patients.
Safety concerns
ELEVIDYS (also known as delandistrogene moxeparvovec or dela mox, and SRP-9001) is a gene therapy for patients with Duchenne muscular dystrophy (DMD) that is produced by Sarepta Therapeutics.
The FDA’s announcement about ELEVIDYS follows it placing Sarepta Therapeutics investigational gene therapy clinical trials for limb girdle muscular dystrophy on hold following three deaths and safety concerns on 18 July. The FDA had said that three deaths appeared to have been a result of acute liver failure in people treated with ELEVIDYS or an investigational gene therapy using the same AAVrh74 serotype that is used in ELEVIDYS.
Distribution of ELEVIDYS
The FDA had also requested on 18 July that Sarepta Therapeutics voluntarily stop all shipments of ELEVIDYS, and Sarepta Therapeutics announced a voluntary pause of ELEVIDYS shipments in the US. Following this new announcement by the FDA on 28 July, Sarepta Therapeutics has said that it will resume shipping ELEVIDYS to sites of care for treatment of ambulatory patients with DMD imminently.
Roche, Sarepta’s global partner in developing ELEVIDYS, said on 23 July that it had decided to voluntarily and temporarily pause all new orders of ELEVIDYS to countries outside the US that reference the FDA for local approval, and in Named Patient Supply (NPS) countries. This pause applies to the ambulatory DMD population, as it previously discontinued treatment of non-ambulatory individuals. Following the FDA’s new announcement on 28 July, Roche has now said that it will immediately resume new orders and shipments of ELEVIDYS for ambulatory patients outside of the US.
ELEVIDYS in Europe
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion regarding ELEVIDYS on 23 July, which means they do not support approval of its conditional marketing authorisation application for the treatment of people with DMD who are ambulatory and aged three to seven years.
European regulators have also requested that Roche and Sarepta temporarily halt clinical studies 104 (NCT06241950), 302 (ENVOL, NCT06128564) and 303 (ENVISION Study 303, NCT05310071). Roche has paused dosing for the ENVISION study outside Europe.
ELEVIDYS in the UK
While ELEVIDYS has not yet been approved as a treatment in the UK, the Medicines and Healthcare products Regulatory Agency (MHRA) does take into account decisions by the FDA and EMA when deciding on whether to approve new treatments in the UK.