Sarepta Therapeutics Begins Dosing Duchenne Patients with Newly Approved Gene Therapy

August 3, 2023

Sarepta Therapeutics has started dosing Duchenne muscular dystrophy patients with its recently approved gene therapy, Elevidys, and plans to serve more in the coming week and a half. The regulatory body has granted approval for the treatment of four- and 5-year-old Duchenne patients, costing $3.2 million. UnitedHealthcare has issued a coverage policy and expects to expand in the next three to six months. Sarepta also stated that uptake is quicker than expected, and they believe it could be accessible to up to 95% of Duchenne patients in the long run. However, sales will build up progressively and rely on a confirmatory trial due in the fourth quarter.

What You Need to Know:

  • U.S. regulators have approved eight gene therapies for inherited diseases, with most approvals happening since the start of 2022.
  • Selling these complex and costly treatments remains challenging due to their high price tags and the lengthy process that some treatments require.
  • Novartis’ spinal muscular atrophy drug, Zolgensma, is proving successful, while others like CSL and UniQure’s hemophilia gene therapy Hemgenix are taking time to gain traction commercially.
  • Sarepta faces hurdles with its therapy Elevidys, which hasn’t proven to improve Duchenne patients’ function and has age restrictions, among other constraints.
  • Sarepta’s CEO, Doug Ingram, points to positive signs, such as activating more than 50 treatment sites.
  • If Sarepta’s trial succeeds, the FDA will loosen Elevidys’ age restrictions; otherwise, it may withdraw the drug from the market.
  • Pfizer could quickly acquire market share with its similar gene therapy; initial Phase 3 results are expected later this year.
  • Sarepta has avoided early revenue forecasts for Elevidys, but there is a strong demand for the therapy.

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[Source: BioPharma Dive, August 3rd, 2023]

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