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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