Genetic medicine company Sarepta Therapeutics Inc (NASDAQ:SRPT) confirmed on Wednesday that the UK Medicines and Healthcare products Regulatory Agency (MHRA) will allow dosing to continue without interruption in the ENVISION study (SRP-9001-303), a global Phase 3 trial of ELEVIDYS (delandistrogene moxeparvovec-rokl) for Duchenne muscular dystrophy.

ENVISION is a randomized, double-blind, placebo-controlled study evaluating ELEVIDYS in non-ambulatory and older ambulatory patients. The decision ensures continued patient access to the only approved gene therapy targeting Duchenne.

ELEVIDYS is a one-time, adeno-associated virus (AAV)-based gene therapy designed to deliver a transgene encoding ELEVIDYS micro-dystrophin to address mutations in the DMD gene. In the United States, it is approved for patients aged four and older with confirmed DMD mutations, including under accelerated approval for non-ambulatory individuals.

Sarepta maintains a leadership position in genetic medicine for rare diseases and is expanding its pipeline across neuromuscular, CNS and cardiac indications.