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Roche : Evrysdi Continues To Improve Motor Function And Survival In Infants With Type 1 SMA

Roche (RHHBY) and PTC Therapeutics Inc. (PTCT) said results from part 2 of the pivotal FIREFISH trial demonstrated that infants with type 1 spinal muscular atrophy or SMA treated with Roche's Evrysdi or risdiplam obtained increases in survival and sustained improvements in achieving key motor milestones. The milestones include head control, sitting, rolling over, and further developing towards acquiring the ability to stand, and walk.

Data from the trial, which is a phase 2/3 global study evaluating Evrysdi in infants aged 1-7 months at enrollment with symptomatic Type 1 spinal muscular atrophy or SMA, demonstrated that at 24 months of treatment, 93% of infants were alive and 83% were alive without permanent ventilation.

There were no new deaths between months 12 and 24. Infants treated with Evrysdi maintained the ability to feed orally 92% at month 24. Further exploratory data suggested similar maintenance in ability to swallow 95%.

In the natural course of the disease, infants with type 1 SMA older than 12 months generally require feeding support. In addition, fewer hospitalizations were observed during the second year of treatment with Evrysdi compared with the natural course of the disease, with 34% not requiring hospitalization during 24 months of treatment.

Additional motor milestones were reached across Part 2 FIREFISH study after 24 months of treatment, the companies said.

Evrysdi is designed to treat SMA by increasing and sustaining the production of the SMN protein, which is found throughout the body and is critical for maintaining healthy motor neurons and movement.

Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. More than 3,000 patients are now treated with Evrysdi in clinical trial, compassionate use and real-world settings.

Evrysdi is marketed in the United States by Genentech, a member of the Roche Group.

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