Clinical Research Informer

Sarepta wins FDA approval of another drug for rare muscular dystrophy

Bio Pharma Dive

FEBRUARY 25, 2021

Like other Sarepta drugs, it comes with a high price tag and its clinical benefits haven't been confirmed. Amondys 45 is now cleared for the estimated 8% of Duchenne patients eligible for treatment.

FDA Approval

FDA Approval Drugs

Sarepta’s Elevidys Reaches Finish Line as First Gene Therapy Approved for Duchenne Muscular Dystrophy

XTalks

JUNE 28, 2023

The approval is restricted to ambulatory patients within this narrow age range due to uncertainty around its effectiveness in older children, which Sarepta hopes to clarify in a confirmatory trial. As such, the agency is requiring Sarepta to confirm the therapy’s clinical benefit as part of the confirmatory trial.

Gene Therapy

Gene Therapy Gene Protein FDA Approval

New Rare Disease Drugs and Research Advancements

XTalks

FEBRUARY 27, 2024

Developed by Sarepta Therapeutics, the therapy is approved for ambulatory children four to five years of age with DMD who have a confirmed mutation in the dystrophin gene. Sarepta hopes to clarify Elvidys’ effectiveness in older children in a confirmatory trial.

Research

Research Drugs Gene Therapy Clinical Trials

Sarepta wins FDA approval of another drug for rare muscular dystrophy

Sarepta’s Elevidys Reaches Finish Line as First Gene Therapy Approved for Duchenne Muscular Dystrophy

New Rare Disease Drugs and Research Advancements

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