The US Food and Drug Administration has approved a new drug for Duchenne muscular dystrophy (DMD) on the basis of data showing that the medicine increased the production of the dystrophin protein in patients – a surrogate for improved motor function. The drug, Vyondys 53, was developed by Sarepta Therapeutics Inc, whose first DMD drug Exondys 51 was approved by the FDA in 2016, also on the basis of a surrogate endpoint.
