First orphan drug approved for Duchenne muscular dystrophy

Release Date: 15-Oct-2016

Food and drug Association (FDA), U.S, has announced Exondys 51 (eteplirsen) injection as the orphan drug for treatment of patients with Duchenne muscular dystrophy (DMD). Exondys 51 has been developed by Sarepta Therapeutics of Cambridge, Massachusetts. It is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD.


DMD is an uncommon genetic disorder characterized by progressive muscle deterioration and paleness. Absence of dystrophin, a protein generally helps in tightening muscle cell. DMD is found to be most common kind of muscular dystrophy. Its occurrence is recorded to be one in every 3,600 male infants worldwide and rare cases in females. The disease causes severe weakness in patients in their early teens followed by life-threatening heart and respiratory conditions during their adulthood.


Exondys 51’s fast track approval is based on the surrogate endpoint of dystrophin increase in skeletal muscle observed in some Exondys 51-treated patients. The most common side effects reported by patients taking Exondys 51 in the clinical trials were balance disorder and vomiting.
FDA grants orphan drug status for rare and life threatening diseases that affect less than 200,000 people per year in the U.S. Also, FDA provide several beneficiaries to drug developers including assistance with clinical study design and drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.


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