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CHMP positive for Evrysdi for the treatment of 5q spinal muscular atrophy.- Roche

Read time: 1 mins
Published:4th Mar 2021
Roche announced that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Evrysdi (risdiplam) for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. SMA is a leading genetic cause of death in infants and 5q SMA is the most common form of the disease. SMA causes progressive muscle weakness and atrophy, and significant unmet need remains, particularly in adults living with this condition. The CHMP recommendation is based on data from two clinical studies designed to represent a broad spectrum of people living with SMA: FIREFISH in symptomatic Type 1 infants aged 2 to 7 months and SUNFISH in symptomatic Type 2 and 3 children and adults aged 2 to 25 years. SUNFISH is the first and only placebo-controlled trial to include adults with Types 2 and 3 SMA. Evrysdi has been approved in seven countries (US, Chile, Brazil, Ukraine, South Korea, Georgia and Russia) and submitted in a further 30 countries.
Condition: Spinal Muscular Atrophy
Type: drug

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