Roche ’s risdiplam shows significant improvement in survival and motor milestones in infants with Type 1 spinal muscular atrophy (SMA)

Basel, 28 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented 1-year data from FIREFISH Part 2, a pivotal global study evaluating risdiplam in infants aged 1 – 7 months old with symptomatic Type 1 spinal muscular atrophy (SMA). The study met its primary endpoint with 29% of infants (12/41; p<0.0001) sitting without support for five seconds by month 12, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). No infants achieve this milestone in the natural history of Type 1 SMA. In addition, 18 (43.9%) infants were able to hold their head upright, 13 (31.7%) were able to roll to the side and 2 (4.9%) infants were able to stand with support, as measured by the Hammersmith Infant Neurological Examination 2 (HINE-2). Safety for risdiplam in the FIREFISH study was consistent with its known safety profile.“These results confirm the clinically meaningful efficacy of risdiplam in infants with an advanced and difficult-to-treat disease,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development. “We thank the SMA community for their partnership and es pecially the 62 families from around the world who participated in Parts 1 and 2 of the FIREFISH study.” The data were selected for the 72nd American Academy of Neurology (AAN) Annual Meeting and will be made available online via virtual presentation in the coming weeks (in lieu of an in-person event). Roc...
Source: Roche Media News - Category: Pharmaceuticals Source Type: news