Decision day for Sarepta’s second Duchenne drug

The FDA is expected to make a decision today on Vyondys 53, a new drug aimed at treating Duchenne muscular dystrophy from Sarepta Therapeutics. If approved, it would expand the population of Duchenne patients eligible for treatment; approval would also come three years after the FDA gave a controversial green light to Sarepta's first Duchenne drug,  Exondys 51.

Vyondys 53 targets an aberration in the gene sequence known as exon 53. In clinical trials, the drug, administered via a weekly infusion, was shown to increase an important muscle protein called dystrophin that is normally missing in children with Duchenne.