Following our request to receive updates about the risdiplam clinical development programme, Roche is delighted to share with us that the U.S. Food and Drug Administration (FDA) has approved risdiplam for the treatment of SMA in adults and children 2 months of age and older. In the U.S., Risdiplam's brand name is Evrysdi™.
The FDA approval is based primarily on data from two studies, which evaluated the efficacy and safety of risdiplam in symptomatic infants with Type 1 SMA aged 2 to 7 months (FIREFISH study) and in people with Types 2 or 3 SMA aged 2 to 25 years (SUNFISH study).
In addition to the FIREFISH and SUNFISH trials that were the basis for the approval in the U.S., the ongoing risdiplam clinical development programme involves more than 450 people with SMA. The programme includes pre-symptomatic infants to adults up to 60 years old; participants with a broad spectrum of symptoms and motor function, as well as patients previously treated with other SMA therapies.
Roche continues to collaborate with health authorities, government agencies and other stakeholders around the world to ensure broad and rapid access to risdiplam to all patients who can benefit from the treatment. As such, Roche has existing regulatory applications under review in Brazil, Chile, China, Indonesia, Russia, South Korea and Taiwan. They hope the U.S. approval serves as a positive signal for future news around the world. In Europe, a Marketing Authorisation Application submission to the European Medicines Agency (EMA) is imminent.