Sarepta wins FDA approval for DMD gene therapy

The US FDA has approved Elevidys (delandistrogene moxeparvovec), Sarepta Therapeutics’s adeno-associated virus-based gene therapy for the treatment of Duchenne muscular dystrophy (DMD).

Having delayed the action date by almost a month, the US Food and Drug Administration (FDA) gave the thumbs up to Elevidys (previously known as SRP-9001) yesterday, a single-dose gene transfer therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with DMD.

Sarepta’s CEO Doug Ingram described the approval as “a watershed moment for the treatment of Duchenne” before acknowledging “the decades of dedication and work from the patient community, families, clinicians, and our Sarepta colleagues that resulted in today’s approval.”

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As part of the Biologics License Application (BLA), the FDA approved manufacture of Elevidys drug substance at Catalent’s facility in Harmans, Maryland, added through the $1.2 billion acquisition of Paragon Bioservices in 2019. The final formulated drug product will be manufactured at Catalent’s site in Baltimore, Maryland.

The contract development and manufacturing organization (CDMO) has expanded the Harmans site to support the progress of SRP-9001, and earlier this year strengthened its deal with Sarepta to prepare for commercial production.

Elevidys joins just a handful of pureplay gene therapies to win approval in the US. The others are: