Risdiplam data from Part 1 of pivotal FIREFISH study show infants with Type 1 spinal muscular atrophy achieve key motor milestones and improved survival after one year of treatment
Basel, 29 April 2019 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new data for its approved and investigational medicines for the treatment of neurological conditions will be presented at the 71st American Academy of Neurology (AAN) Annual Meeting from 4-10 May in Philadelphia, PA. Presentations include data from a pivotal study for risdiplam in spinal muscular atrophy (SMA), which has the potential to become the first oral treatment for this community.
Spinal Muscular Atrophy (SMA)
New data will be presented for risdiplam, an investigational oral survival motor neuron 2 (SMN2) splicing modifier for SMA, which is designed to increase and sustain SMN protein levels in the central nervous system (CNS) and throughout the body. Platform presentations include one-year data from the dose-finding Part 1 of the pivotal FIREFISH study on key motor milestones, motor function and survival in infants with Type 1 SMA. Updated safety, tolerability and pharmacokinetics / pharmacodynamics (PK/PD) data, as well as an exploratory efficacy analysis from Part 1 of the pivotal SUNFISH study in people aged 2-25 years with SMA Types 2 or 3 will also be presented.