Risdiplam showed significant improvement in adults with SMA type 2,3

Roche publishes its latest results from Pivotal SUNFISH Part 2 with Risdiplam. Another milestone is the treatment of adult patients with spinal muscular atrophy type 2 and 3.

Roche’s risdiplam showed significant improvement in motor function in people
aged 2-25 with type 2 or 3 spinal muscular atrophy
• First placebo-controlled trial to include adults with SMA demonstrates risdiplam improved or
stabilised motor function
• Medically meaningful and statistically significant results in primary and key secondary
• Pivotal SUNFISH Part 2 study population represents broad, real-world spectrum of people living with SMA

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Risdiplam demonstrated statistically significant and medically meaningful motor milestone improvement in infants with Type 1 SMA

Basel, 23 January 2020 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced positive topline results from the pivotal Part 2 of the FIREFISH study, evaluating risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA).

The primary outcome measure of the study was the proportion of infants sitting without support for at least five seconds at 12-months of treatment, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). Safety for risdiplam in the FIREFISH study was consistent with its known safety profile and no new safety signals were identified. To date, more than 400 patients have been treated with risdiplam across all studies, with no treatment related safety findings leading to study withdrawal in any risdiplam trial

Material provided Roche

NCT02913482 (Firefish)

Roche announced that FDA grants priority review to risdiplam for spinal muscular atrophy

Wie start the week with a great news!

Roche announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) and granted Priority Review for risdiplam, an investigational survival motor neuron-2 (SMN-2) splicing modifier for SMA. Risdiplam is designed to increase and sustain SMN protein levels both throughout the central nervous system and peripheral tissues of the body. The FDA is expected to make a decision on approval by May 24, 2020.

“The FIREFISH and SUNFISH trials were designed to represent the real world spectrum of people living with SMA and include many people previously underrepresented in clinical trials,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “We look forward to working closely with the FDA to explore broad access to risdiplam for all individuals in the community who might benefit.”