Sarepta Receives the US FDA's Approval for VYONDYS 53 (golodirsen) Injection to Treat Duchenne Muscular Dystrophy (DMD) in Patients with Amenable to Skipping Exon 53
- Sarepta’s VYONDYS 53 is an antisense oligonucleotide derived using phosphorodiamidate morpholino oligomer (PMO) platform targeting DMD patients in patients with a confirmed mutation amenable to exon 53 skipping
- In Aug 2019- the company received a CRL for VYONDYS 53 from Drug Evaluation 1 following the New Drug Application (NDA) submission to and review by the Division of Neurology Products (the Review Division)
- VYONDYS 53 is approved under accelerated review based on increase in dystrophin production in skeletal muscle of patients amenable to exon 53 skipping and will be commercialized in the US and has also demonstrated safety and effectiveness
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