Tags : Risdiplam

Chugai Reports NDA Submission of Risdiplam to the MHLW as

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 or 3 FIRESISH study results: improvement in survival and motor milestones in infants. SUNFISH study results: improvement in motor function in people aged 2-25 with […]Read More

PTC Reports the EMA’s Acceptance of MAA for Evrysdi (risdiplam)

Shots: The MAA submission is based on dose-finding Part 1 and confirmatory Part 2 of the FIREFISH and SUNFISH studies evaluating the efficacy and safety of Evrysdi (risdiplam) in symptomatic infants with type 1 SMA aged 2-7mos. and in people with types 2/3 SMA aged 2-25 yrs/ respectively The submission also includes data from JEWELFISH […]Read More

Royalty Pharma Acquires PTC’s Royalty Interest in Risdiplam for $650M

Shots: PTC Therapeutics to receive $650M as upfront in cash in exchange for ~43% of the risdiplam royalties, up to a specified amount while PTC maintains the majority of the risdiplam royalty stream and retains all economics associated with up to ~$400M in remaining regulatory and commercial milestones The collaboration enables PTC to further develop […]Read More

The US FDA Delays its Decision on Roche’s Risdiplam for

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 of additional data, including SUNFISH Part 2 study The company has submitted filing applications for in seven countries with submission in China imminent and currently […]Read More

Roche Reports Results of Risdiplam in SUNFISH Part 2 Study

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 its 1EPs & 2EPs i.e. greater change from baseline in MFM-32 & improvement in RULM @12mos., strongest responses in MFM-32 in younger patients aged 2-5 […]Read More

Roche Reports Results of Risdiplam in FIREFISH Study for Infants

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 sitting without support for at least 5sec @12mos. of treatment, assessed by the Gross Motor Scale of BSID-III, consistent safety profile with no new safety […]Read More

Roche Reports the US FDA’s Acceptance of NDA for Risdiplam

Shots: The NDA submission is based on FIREFISH Part-1 and SUNFISH Part-2 studies. The FIREFISH (Part 1 & 2) study involves assessing Risdiplam in 21 & 41 infants aged 1-7mos. with SMA, followed by an open-label extension respectively The SUNFISH (Part 1 & 2) study involves assessing Risdiplam vs PBO in children & young adults […]Read More

Roche Reports Results of Risdiplam in Pivotal SUNFISH Study for

Shots: The pivotal part 2 SUNFISH study involves assessing Risdiplam vs PBO in patients aged 2-25 yrs. with type 2 or 3 spinal muscular atrophy The SUNFISH study resulted in meeting its 1EPs i.e, change in the Motor Function Measure 32 (MFM-32) scale @12mos. with consistent safety profile and no new signals were identified Risdiplam […]Read More