Validation of the MAA confirms that the submission is accepted and begins the formal scientific evaluation process by EMA’s Committee for Medicinal Products for Human Use (CHMP). Following our request, Roche provided us with a community statement to announce the news. The EMA has granted the marketing application with accelerated assessment, which means that the evaluation process can be reduced from 15 to 9 months, if the accelerated assessment timetable is maintained.

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.