Roche?s risdiplam meets primary endpoint in pivotal SUNFISH trial in people with type 2 or 3 spinal muscular atrophy
[caption id="attachment_9277" align="aligncenter" width="747"] Press Release[/caption]
- Study demonstrated statistically significant improvements in the overall study population with Type 2 or 3 SMA
- No treatment related safety findings leading to withdrawal seen in any risdiplam trial to date
- Data will be shared with health authorities globally, including the U.S. Food and Drug Administration (FDA)
- SUNFISH (NCT02908685) ? as above. Results will be presented at an upcoming medical congress.
- 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). Part 2 is ongoing.
- 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 is currently recruiting.
- 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.