Genentech, a member of the Roche Group, announced new interim data from two studies of Evrysdi® (risdiplam)—JEWELFISH and RAINBOWFISH.
Data from JEWELFISH, an ongoing open-label study primarily evaluating the safety of Evrysdi in people aged 1 to 60 years who have been previously treated with another SMA-targeting therapy (i.e., nusinersen, onasemnogene abeparvovec) showed the safety profile of Evrysdi and increase in SMN protein levels are consistent with those observed in other Evrysdi studies.
Preliminary efficacy data from RAINBOWFISH, an ongoing open-label study evaluating Evrysdi in babies from birth to 6 weeks with pre-symptomatic SMA, showed that infants treated for 12 months achieved age-appropriate motor milestones, including sitting, standing, and walking, and improvements in motor function.
Evrysdi is based on PTC Therapeutics (PTC) science and is commercialized in the U.S. by Genentech. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC.
PTC Therapeutics (PTC) announced in March that the European Commission (EC) granted marketing authorization to Evrysdi™ (risdiplam) for the treatment of spinal muscular atrophy (SMA) in patients 2 months and older. Evrysdi will be for SMA Type 1, Type 2 or Type 3 patients with one to four SMN2 copies.
PTC also recently announced that a $20 million milestone payment was triggered by the first commercial sale of Evrysdi in the European Union under its License and Collaboration Agreement with Roche
Accredo is the only specialty pharmacy (SP) that prepares and ships Evrysdi directly to patients each month. Evrysdi is not available at retail pharmacies.
Accredo will call patients to welcome them to the program and to schedule delivery times that work best for them. It is important for patients to answer any calls from Accredo to prevent any delays in treatment.
Accredo is a specialty pharmacy that serves patients with complex and chronic health conditions, including cancer, hepatitis C, HIV, bleeding disorders and multiple sclerosis. Accredo delivers a level of expertise and care through 15 condition-specific Therapeutic Resource Centers (TRC).
Takeaway: Evrysdi is based on PTC Therapeutics (PTC) science and is commercialized in the U.S. by Genentech. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC