We received a great pressrelease from Roche. Risdiplam is a milestone ahead.
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 EvrysdiTM (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.