Elevidys (delandistrogene moxeparvovec-rokl) is an adeno-associated virus (AAV)-based gene therapy indicated for the treatment of ambulatory paediatric patients, aged four to five years, with a confirmed mutation in the DMD gene
FDA approves Sarepta's gene therapy Elevidys. (Credit: Myriams-Fotos from Pixabay)
US-based biotechnology company Sarepta Therapeutics has received the US Food and Drug Administration (FDA) accelerated approval for Elevidys to treat a type of duchenne muscular dystrophy (DMD).
Elevidys (delandistrogene moxeparvovec-rokl) is an adeno-associated virus (AAV)-based gene therapy that addresses the underlying cause of Duchenne muscular dystrophy, the mutations in the dystrophin gene.
It works through the targeted production of a gene that codes for a shortened form of dystrophin to muscle cells, known as Elevidys micro-dystrophin to treat DMD.
The drug is indicated for ambulatory paediatric patients, aged four to five years, with a confirmed mutation in the DMD gene.
The US FDA approved the drug under its accelerated approval pathway, and continued approval is based on the verification of clinical benefit in confirmatory trials.
Elevidys has been evaluated in three ongoing clinical studies, SRP-9001-101, SRP-9001-102 and SRP-9001-103.
The FDA accelerated approval was supported by efficacy data from studies, SRP-9001-102 and SRP-9001-103 and safety data from all three trials.
In the clinical studies, patients treated with Elevidys experienced acute serious liver injury, immune-mediated myositis and myocarditis.
Vomiting, nausea, liver function test increase, pyrexia and thrombocytopenia are the most common adverse reactions in clinical studies.
Furthermore, Sarepta intends to complete its Phase 3 EMBARK study, as a confirmatory trial, with top-line results expected later this year.
Sarepta president and CEO Doug Ingram said: “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.
“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.”