The FDA has approved an exon-skipping antisense drug which is set to rival Sarepta’s Vyondys 53.
Japanese pharma, N S Pharma, a wholly owned subsidiary of Nippon Shinyaku Co., received marketing approval for its PMO drug, Viltepso (viltolarsen), in the treatment of DMD patients with a mutation amenable to exon 53 skipping. Patients with this mutation, the same targeted by Sarepta’s Vyondys 53, represent approximately 8% of the total estimated 10,000 DMD population in the U.S.
Approval for Viltepso was based on two small clinical trials with a total of 32 patients tested. In one of the two studies, the drug increased dystrophin production, with levels increasing on average by 0.6% at baseline to 5.9% at week 25. While Viltepso helped patients create more dystrophin than seen in the Vyondys 53 clinical trials, the average increase to 1.02% of normal dystrophin production after 48 weeks treatment with Vyondys 53 was from just 0.1% at base line. Therefore, the change in dystrophin levels seen in the two drugs was similar, at about tenfold.
Prior to its US regulatory approval, Viltepso was granted Priority Review, Rare Paediatric Disease, Orphan Drug and Fast Track designations by the FDA, along with Japanese regulatory approval in March 2020.
N S Pharma is yet to announce pricing. Both of Sarepta’s DMD drugs (Exondys 51 and Vyondys 53) are priced based on weight, with a cost of approximately US$300,000 p.a. for a 20 kg child.
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