Sarepta wins FDA approval for DMD gene therapy

The US FDA has approved Elevidys (delandistrogene moxeparvovec), Sarepta Therapeutics’s gene therapy for Duchenne muscular dystrophy (DMD).

Dan Stanton, Managing editor

June 23, 2023

2 Min Read
Sarepta wins FDA approval for DMD gene therapy
Image: DepositPhotos/ Premium_shots

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.”


Image: DepositPhotos/

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:

  • October 2015: BioVex/Amgen’ Imlygic (talimogene laherparepvec)

  • December 2017: Spark Therapeutics/Roche’s Luxturna (voretigene neparvovec)

  • May 2019: AveXis/Novartis’ Zolgensma (onasemnogene abeparvovec)

  • August 2022: Bluebird bio’s Zynteglo (betibeglogene autotemcel, or beti-cel)

  • September 2022: Bluebird bio’s Skysona (elivaldogene autotemcel, or eli-cel)

  • November 2022: CSL Behring’s Hemgenix (etranacogene dezaparvovec)

  • December 2022: Ferring Pharmaceuticals’ Adstiladrin (nadofaragene firadenovec)

About the Author(s)

Dan Stanton

Managing editor

Journalist covering the international biopharmaceutical manufacturing and processing industries.

Founder and editor of Bioprocess Insider, a daily news offshoot of publication Bioprocess International, with expertise in the pharmaceutical and healthcare sectors, in particular, the following niches: CROs, CDMOs, M&A, IPOs, biotech, bioprocessing methods and equipment, drug delivery, regulatory affairs and business development.

From London, UK originally but currently based in Montpellier, France through a round-a-bout adventure that has seen me live and work in Leeds (UK), London, New Zealand, and China.

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