Data for Roche ’s Evrysdi® (risdiplam) published in New England Journal of Medicine shows significant improvement in survival and motor milestones in babies with Type 1 spinal muscular atrophy (SMA)

Basel, 29 July 2021 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the New England Journal of Medicine (NEJM) has published data from FIREFISH Part 2, a pivotal global study evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies aged 1-7 months old with symptomatic Type 1 spinal muscular atr ophy (SMA). The study met its primary endpoint with 29% of infants (12/41) sitting without support for at least five seconds* by month 12, a milestone not seen in the natural course of the disease. Safety for Evrysdi in the FIREFISH Part 2 study was consistent with its known safety profile.“Without treatment, babies with Type 1 SMA are unlikely to survive beyond two years of age,” said Professor Laurent Servais, M.D., Ph.D., FIREFISH investigator and Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre. “Important motor milestones, such as sitti ng, rolling over and swallowing, are the fundamental building blocks that can help these babies achieve optimal outcomes with Evrysdi, potentially reducing the need for ventilation and increasing the rate of survival.’At the time of the data analysis, the median duration of treatment with Evrysdi was 15.2 months and the median age was 20.7 months. At month 12, 93% (38/41) of infants were alive and 85% (35/41) were free from permanent ventilation. Without treatment, the median age of death or permanent ventilation was 13.5 months in a natural history cohort...
Source: Roche Media News - Category: Pharmaceuticals Source Type: news

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Source: Annals of Medicine - Category: Internal Medicine Authors: Source Type: research
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Source: Monaldi Archives for Chest Disease - Category: Respiratory Medicine Authors: Source Type: research
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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
Source: Roche Media News - Category: Pharmaceuticals Source Type: news
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