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

Written by Customer Service on . Posted in Public Companies.

FIREFISH Part 2 study met its primary endpoint by demonstrating a significant increase in motor milestones in infants aged 1-7 months after 12 months of treatmentLarge, pivotal global study confirms clinically meaningful efficacy seen in the dose-finding Part 1 of the trialSafety was consistent with the safety profile observed to date and no new safety signals were identifiedBasel, 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.
Roche Group Media Relations
 Phone: +41 61 688 8888 / e-mail: media.relations@roche.com
– Nicolas Dunant (Head)
– Patrick Barth
– Daniel Grotzky
– Karsten Kleine AttachmentRoche media release_risdiplam_28042020

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