FDA Raises Concerns About Golodirsen as Potential Exon 53 Skipping Duchenne Therapy
The U.S Food and Drug Administration (FDA) has rejected Sarepta Therapeutic’s application seeking accelerated approval of golodirsen (SRP-4053) for Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping. Golodirsen (SRP-4053) was placed under priority review by the FDA in February 2019. The FDA’s complete response…