Sarepta Therapeutics has announced a strategic restructuring plan designed to prioritise high-value, high impact programmes, and support its long-term financial viability. This plan includes a 36% workforce reduction, impacting approximately 500 employees, and an emphasis on near- and mid-term opportunities from the siRNA platform. Several programmes, including most of the… Read More
The US Food and Drug Administration (FDA) has expanded its approval for Elevidys (delandistrogene moxeparvovec-rokl) to include individuals with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least four years of age. Confirming the functional benefits, the FDA granted traditional approval for ambulatory… Read More
Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD. It is approved… Read More
The US Food and Drug Administration (FDA) Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) have voted eight to six in favour of a new gene therapy for Duchenne muscular dystrophy. The vote supports the accelerated approval of Sarepta Therapeutics’ SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulatory patients with… Read More
