Roche receives positive CHMP opinion for Evrysdi, the first and only at home spinal muscular atrophy (SMA) treatment with proven efficacy in adults, children and infants two months and older

Basel, 26 February 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today 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 was completed under the accelerated assessment pathway, which is offered to medicines deemed to be of major interest for public health and therapeutic innovation. A final decision regarding approval is expected from the European Commission in the next two months and will be applicable to all 27 European Union member states, as well as Iceland, Norway, and Liechtenstein. Evrysdi was approved by the U.S. Food and Drug Administration (FDA) in August 2020.“Our close partnership with the SMA community has enabled the development of the first ‘at-home’ treatment for SMA in infants, children and adults with varying levels of disease severity, the majority of whom remain untreated,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Office r and Head of Global Product Development....
Source: Roche Investor Update - Category: Pharmaceuticals Source Type: news

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