September 19, 2016
Eteplirsen (Exondys 51) injection received US Food and Drug Administration (FDA) approval today under the accelerated approval pathway for the treatment of patients with Duchenne muscular dystrophy (DMD).
This is the first FDA-approved drug to treat individuals with the rare genetic disorder. DMD, a form of muscular dystrophy, is marked by progressive muscle weakness and difficulty walking. Patients with DMD may also experience fatigue, learning difficulties, and intellectual disability. Symptoms of DMD first begin in childhood.
According to an FDA statement, eteplirsen is indicated for those with “a confirmed mutation of the dystrophin gene amenable to exon 51 skipping,” which approximately 13% of patients with DMD have. The dystrophin gene is the largest human gene.
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The FDA made its decision to approve the drug based on the surrogate endpoint of dystrophin increase in skeletal muscle that was experienced by some individuals who were treated with eteplirsen.
The most common side effects are balance disorder and vomiting.
The accelerated approval pathway allows for the approval of drugs for serious or life-threatening conditions that provide a significant advantage over other existing treatments, according to the statement.
With this type of approval, patients will gain access to the drug while Sarepta Therapeutics, the manufacturer of eteplirsen, conducts a clinical trial to confirm the drug’s clinical benefit.
The fast track designation allows for an expedited process for the development and review of medications that meet unmet medical needs.
-Meredith Edwards White