Category: Evrysdi (risdiplam)
Shots: The approval is based on two studies i.e. FIREFISH & SUNFISH. The FIREFISH study involves assessing Evrysdi in infants aged 2-7mos. with symptomatic Type 1 SMA, and the SUNFISH study assesses Evrysdi in children & young adults aged 2-25yrs. with Type 2 or 3 SMA Evrysdi demonstrated improvement in motor function, 29.3% of participants […]
Shots: The 2-year data from Part 2 of P-II/III FIREFISH study involves assessing Evrysdi in 41 infants aged 1-7mos. with symptomatic Type 1 SMA Results: improvement in maintained motor function b/w 12-24mos., babies were able to sit without support for at least 5sec. after 24mos. vs 12mos. (61% vs 29%), 92% maintained the ability to […]
Shots: Genentech reports the new 2yrs. data from part 2 of SUNFISH study assessing Evrysdi vs PBO in people aged 2-25 years with Type 2 or Type 3 SMA Results: improvement in maintained motor function b/w 12-24mos. as measured by MFM-32; change from baseline in SMAIS score; stabilized motor function after 12mos. as per MFM-32, […]
Shots: Roche has reported the data from the dose-finding Part 1 of the FIREFISH study assessing Evrysdi in patients aged 1-7mos. with Type 1 SMA. Part 1 evaluated several doses of Evrysdi and determined the therapeutic dose of 0.2 mg/kg for Part 2 Part 1 data demonstrated that 99% of infants survive without permanent ventilation […]