Roche’s Evrysdi continues to improve motor function and survival in babies with Type 1 Spinal Muscular Atrophy (SMA)

• More than twice as many babies (61% vs. 29%) were able to sit without support for at least five seconds after 24 months compared to 12 months of treatment
• Evrysdi increased survival and reduced need for permanent ventilation
• Evrysdi has proven efficacy across adults, children and babies 2 months and older
  
  

Basel, 15 April 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new 2-year data from Part 2 of FIREFISH, a Phase 2/3 global study evaluating Evrysdi (risdiplam) in infants aged 1-7 months at enrollment with symptomatic Type 1 spinal muscular atrophy (SMA). The data showed Evrysdi continued to improve motor function between months 12 and 24, including the ability to sit without support. The study also showed Evrysdi continued to improve survival, improve ability to feed orally and reduce the need for permanent ventilation*. Exploratory data suggested Evrysdi continued to improve the ability to swallow and reduce hospitalisations compared to the natural course of Type 1 SMA. Safety for Evrysdi was consistent with its established safety profile. These longer-term data build upon one-year pivotal findings from FIREFISH Part 2 and will be presented at the 73rd American Academy of Neurology (AAN) Annual Meeting being held virtually April 17-22, 2021.

“The natural course of Type 1 SMA shows us that, sadly, without treatment children are never able to sit without support and typically don’t survive beyond the age of two,” said FIREFISH investigator Dr. Basil Darras, Professor of Neurology at Harvard Medical School and Director of the Spinal Muscular Atrophy Program at Boston Children's Hospital. “It is encouraging to see that infants continued to improve after 12 months of treatment, with twice as many of those who received Evrysdi for two years able to sit without support for at least five seconds. Infants treated with Evrysdi also experienced a range of improvements in motor function abilities, a reduction in serious events typically caused by disease progression, such as the need for permanent ventilation or hospitalisation, and increased rate of survival.”

The primary endpoint of the study was the percentage of infants able to sit without support for at least five seconds at month 12. At 12 months, infants treated with Evrysdi demonstrated improved ability to sit without support for at least five and 30 seconds. Twenty-four month data showed continued improvements from month 12, with 61% (25/41) vs. 29% (12/41) able to sit without support for at least five seconds and 44% (18/41) vs. 17% (7/41) able to sit without support for at least 30 seconds, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). Importantly, infants treated with Evrysdi maintained the ability to feed orally (92%; 35/38) at month 24. Further, exploratory data suggest similar maintenance in ability to swallow (95%; 36/38). In the natural course of the disease, infants with Type 1 SMA older than 12 months generally require feeding support.