NS Phrama’s Viltepso (viltolarsen) Receives the US FDA’s Approval for Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping Therapy

Shots:

• The approval is based on P-II two period study; Study 1 includes patients aged 4-10yrs. conducted in North America and Study 2 includes boys aged 5-18yrs. conducted in Japan. The continued approval of VILTEPSO may contingent on confirmation of clinical benefit in P-III confirmatory trial
• Results: patients receiving the dose of (80 mg/kg/wk), 100% patients showed increase in dystrophin levels; 88% patients showed dystrophin levels of 3% or greater than normal; after 20-24wks. of treatment, increment in dystrophin expression (6% vs 0.6%)
• Viltepso has received PR designation, RPD, FT & ODD, prior to its FDA’s approval. In Mar’2020, it has received MHLW’s approval, prior to which it received SAKIGAKE, ODD and designation of conditional early approval system

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