Roche announces 2-year risdiplam data from SUNFISH and new data from JEWELFISH in infants, children and adults with spinal muscular atrophy (SMA)

Basel, 12 June 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented two-year data from Part 1 of its pivotal SUNFISH trial in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA) at the virtual Cure SMA Annual Conference, 8-12 June, 2020. The results of an exploratory efficacy analysis show risdiplam significantly improved motor function after 24 months of treatment compared to natural history data. In addition, preliminary 12 month data from JEWELFISH, a trial in people with all types of SMA aged 6 months to 60 years previously treated with other SMA therapies, showed that treatment with risdiplam led to rapid and sustained increases in SMN protein levels. No new safety signals were observed and the overall adverse event profile was consistent with that of treatment-naive patients.“These 24 month exploratory data are important as they are consistent with the medically meaningful results we saw after one year in Part 2 of the SUNFISH study, designed to represent a broad, real-world SMA population,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development. “We are also encouraged to see an increase in SMN protein levels across both the Sunfish Part 1 and Jewelfish studies. These data reinforce the potential of risdiplam to make a real difference in the lives of the many people living with SMA.” SUNFISH is a large (n=231) global two-part study in children and adults. The dose-findi...
Source: Roche Media News - Category: Pharmaceuticals Source Type: news

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