Sarepta Therapeutics announced U.S. Food and Drug Administration (FDA) accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl), an adeno-associated virus based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. ELEVIDYS is a one-time treatment designed to treat the underlying genetic cause of Duchenne.
“The approval of ELEVIDYS is a watershed moment for the treatment of Duchenne. ELEVIDYS is the first and only gene therapy approved for Duchenne, and this approval brings us closer to our goal of bringing forward a treatment that provides the potential to alter the trajectory of this degenerative disease,” said Doug Ingram, president and chief executive officer, Sarepta. “As we prepare to launch ELEVIDYS, we should acknowledge and celebrate the decades of dedication and work from the patient community, families, clinicians, and our Sarepta colleagues that resulted in today’s approval. Our confirmatory trial, EMBARK, should read out in the fourth quarter of this year. If EMBARK confirms the benefits seen in our prior trials, Sarepta will move rapidly to submit a BLA supplement to expand the approved label as broadly as good science permits.”
Please see the full press release here
Do you want to know more?
- To find out more about Duchenne science, gain crucial knowledge and support, please join us on our Science on Tour workshops. We are coming to 30 locations across the UK in 2023 – book your FREE place here now.
- SAVE THE DATE: Action Duchenne’s International Conference 2023 will be held on the 10th and 11th November and is an amazing opportunity to meet with those involved in every aspect of Duchenne, from families, clinicians, researchers and pharmaceutical companies.
Join National Director Florence Boulton as part of our Vitality London 10,000 Team!
