Roche presents encouraging 1 year FIREFISH study data

Roche’s FIREFISH Part 2 study evaluating risdiplam in 1-7 month old infants with symptomatic Type 1 spinal muscular atrophy met its primary endpoint with 29% of infants sitting without support for five seconds by month 12, (as assessed by the Gross Motor Scale of BSID-III), 43.9% holding their head upright, 31.7% rolling to the side and 4.9% standing with support (as measured by HINE-2). The median duration of treatment at this point was 15.2 months and the median age was 20.7 months. 93% of infants were alive and 85.4% were event-free. The median age of death or permanent ventilation without treatment was 13.5 months in a natural history cohort. Three infants had fatal complications of their disease within the first three months of treatment, but none were considered to be related to risdiplam. 90% had a CHOP-INTEND score increase of at least 4 points, and 56% had a score over 40 (median increase was 20 points). In an exploratory endpoint, 95% of infants who were alive at 12 months could still swallow and 89% could feed orally compared to all infants with Type 1 SMA older than 12 months requiring feeding support in a natural history cohort. Safety for risdiplam in the FIREFISH study was consistent with its previously reported safety profile and no new safety signals were identified.

FIREFISH investigator and Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre, Professor Laurent Servais, said: “These results are particularly encouraging given the median age at enrolment was 5.3 months, so these infants already had progressed disease. Maintaining the ability to swallow is particularly important as it helps infants to feed and suggests risdiplam has a major effect on bulbar function.”

Roche’s Chief Medical Officer and Head of Global Product development, Levi Garraway, said: “These results confirm the clinically meaningful efficacy of risdiplam in infants with an advanced and difficult-to-treat disease. We thank the SMA community for their partnership and especially the 62 families from around the world who participated in Parts 1 and 2 of the FIREFISH study.”