12 December 2019 - The U.S. FDA today granted accelerated approval to Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. 

It is estimated that about 8% of patients with Duchenne muscular dystrophy have this mutation.

The accelerated approval of Vyondys 53 is based on the surrogate endpoint of an increase in dystrophin production in the skeletal muscle observed in some patients treated with the drug. The FDA has concluded that the data submitted by the applicant demonstrated an increase in dystrophin production that is reasonably likely to predict clinical benefit in patients with DMD who have a confirmed mutation of the dystrophin gene amenable to exon 53 skipping. A clinical benefit of the drug, including improved motor function, has not been established. In making this decision, the FDA considered the potential risks associated with the drug, the life-threatening and debilitating nature of the disease and the lack of available therapy.

Read FDA press release