EMA validates the Marketing Authorisation Application (MAA) for risdiplam for the proposed use in people living with SMA
The risdiplam MAA filing is based on data from the FIREFISH and SUNFISH studies which evaluate the efficacy and safety of risdiplam in symptomatic infants with Type 1 SMA aged 2 to 7 months and in people with Types 2 or 3 SMA aged 2 to 25 years, respectively. In addition, the submission incorporates safety data from JEWELFISH, a trial in people with all types of SMA aged 1 to 60 years, previously treated with other SMA therapies.
Risdiplam was granted PRIME (PRIority MEdicines) designation by the EMA in 2018 and Orphan Drug Designation in 2019. In addition to filing in Europe, Roche has existing regulatory applications under review in Australia, Brazil, Canada, Chile, China, Indonesia, Russia, South Korea and Taiwan.
More information
- About risdiplam
- The community update – 17th August 2020
- The press release – 17th August 2020