Shots: The approval is based on the results from two studies i.e. FIREFISH & SUNFISH. The FIREFISH study involves assessing Evrysdi (dry syrup, 60mg) in infants with symptomatic Type 1 […]readmore
Tags : Spinal Muscular Atrophy
Shots: The new interim data from two studies i.e JEWELFISH open-label study evaluating Evrysdi in people aged 1 to 60 yrs previously treated with another SMA-targeting therapy & RAINBOWFISH study […]readmore
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 […]readmore
Shots: The approval is based on two studies i.e. FIREFISH and SUNFISH. The FIREFISH study involves assessing Evrysdi in infants aged 2-7mos. with symptomatic Type 1 SMA, and the SUNFISH […]readmore
Shots: The NDA submission is based on FIREFISH study assessing Risdiplam in infants with symptomatic SMA Type 1 & SUNFISH study in children and young adults with SMA Type 2 […]readmore
Shots: The EC’s approval is based on P-III STR1VE-US and P-I START studies assessing the efficacy & safety of a one-time IV infusion of Zolgensma in symptomatic SMA Type 1 […]readmore
Shots: The US FDA has extended the PDUFA date for its review of risdiplam’s NDA with the expected decision by Aug 24, 2020. The extension is based on the submission […]readmore
Regulatory
Novartis’ Zolgensma (onasemnogene abeparvovec) Receives MHLW’s Approval for Spinal Muscular
Shots: The approval is based on P-I START, START long-term follow-up, P-III STR1VE-US, P-III SPR1NT and P-I/II STRONG (intrathecal). START and STR1VE-US assess Zolgensma (one-time, IV infusion) in symptomatic SMA […]readmore
Shots: The SUNFISH part 2 study involves assessing risdiplam vs PBO in patients aged 2-25yrs. with non-ambulatory type 2 or 3 SMA The SUNFISH part 2 study resulted in meeting […]readmore
Shots: The FIREFISH study involves assessing of Risdiplam in infants aged 1-7 mos. with Type 1 SMA The P-II FIREFISH study results: met its 1EPs i.e, the proportion of infants […]readmore