Genentech’s Evrysdi (risdiplam) Receives the US FDA’s Approval for the Treatment of Spinal Muscular Atrophy in Babies Aged Under Two Months
Shots:
- The approval was based on interim efficacy & safety data from the (RAINBOWFISH) study evaluating the efficacy, safety, PK & PD of Evrysdi in 25 patients aged birth to 6wks. with SMA
- The results showed that patients treated with Evrysdi were able to sit (100%) @1yr., 67% can stand, 50% can walk independently & infants were alive without permanent ventilation @12mos.
- The Evrysdi prescription information has also been revised as part of the label extension including 2yr. pooled results from parts 1 & 2 of the (FIREFISH) trial which showed that 60% & 28% of infants were able to sit & stand without support for 5sec., 40% for 30sec. in symptomatic babies aged 1-7mos. @2yr. at the recommended dose (n=58)
Ref: Genentech | Image: Genentech
Click here to read the full press release
Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.
