Sarepta Therapeutics receives FDA approval for Exondys 51 to treat DMD

• US-based biopharmaceutical company Sarepta Therapeutics has secured the US Food and Drug Administration (FDA) accelerated approval for its Exondys 51 (eteplirsen) as a once-weekly intravenous infusion of 30mg per kg to treat Duchenne muscular dystrophy (DMD).
• Exondys 51 uses Sarepta’s patented phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. Exon skipping enables production of an internally truncated dystrophin protein.
• It binds to exon 51 of dystrophin pre-mRNA, resulting in the removal of this exon during Messenger RNA (mRNA) processing in patients with genetic mutations that are amenable to exon 51 skipping.
• FDA Center for Drug Evaluation and Research director Janet Woodcock said: “Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease.
• “In rare diseases, new drug development is especially challenging due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders.
• “Accelerated approval makes this drug available to patients based on initial data, but we eagerly await learning more about the efficacy of this drug through a confirmatory clinical trial that the company must conduct after approval.”
• DMD is a rare genetic disorder causing progressive muscle deterioration and weakness due to the absence of dystrophin, a protein which helps to keep muscle cells intact.
• Exondys 51 was granted an accelerated approval on the basis of data, which suggested an increased dystrophin production in skeletal muscle.
• Sarepta has been recommended by the FDA to undertake a clinical trial to test the drug’s efficacy in improving motor functions in DMD patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping.

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