Roche provides regulatory update on risdiplam for the treatment of spinal muscular atrophy (SMA)
- U.S. Food and Drug Administration (FDA) extends review time for risdiplam following agreed submission of additional data, including SUNFISH Part 2
- Roche has submitted filing applications for risdiplam in seven countries with submission in China imminent
- Risdiplam is being investigated in infants, children and adults with Type 1, 2 or 3 SMA
- SUNFISH (NCT02908685) ? SUNFISH is a two part, double-blind, placebo controlled pivotal study in people aged 2-25 years with Types 2 or 3 SMA. Part 1 (n=51) determined the dose for the confirmatory Part 2. Part 2 (n=180) evaluated motor function using total score of Motor Function Measure 32 (MFM-32) at 12 months. MFM-32 is a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA. The study met its primary endpoint.
- FIREFISH (NCT02913482) ? an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose-escalation study in 21 infants. The primary objective of Part 1 was to assess the safety profile of risdiplam in infants and determine the dose for Part 2. Part 2 is a pivotal, single-arm study of risdiplam in 41 infants with Type 1 SMA treated for 24 months, followed by an open-label extension. Enrolment for Part 2 was completed in November 2018. The primary objective of Part 2 is to assess efficacy as measured by the proportion of infants sitting without support after 12 months of treatment, as assessed in the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development ? Third Edition (BSID-III) (defined as sitting without support for 5 seconds).
- JEWELFISH (NCT03032172) ? an open-label exploratory trial in people with SMA aged 6 months?60 years who have been previously treated with SMA-directed therapies. The study has completed recruitment.RAINBOWFISH (NCT03779334) ? an open-label, single-arm, multicentre study, investigating the efficacy, safety, pharmacokinetics and pharmacodynamics of risdiplam in babies (~n=25), from birth to six weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. The study is currently recruiting.