Firefish Study in SMA Typ I. Babies started treatment at nearly seven months old
11 (64.7%) infants were able to sit (with or without support) while 9 (52.9%) achieved upright head control after 12 months of treatment as assessed by the Hammersmith Infant Neurological Examination Module 2 (HINE-2). Finally, 1 infant (5.9%) achieved the milestone of standing (supports weight) by this 12-month time point.
Sunfish Study in Patient (2-25 age) in SMA Typ II-III
This scale is designed to detect motor function changes in a broad range of patients, from weak Type 2 to strong Type 3 SMA, and is therefore more appropriate for the SUNFISH population. One patient withdrew from the trial during the open-label extension. Among the patients for which the MFM32 scale has been completed at all visits up to month 12 (n=43), 58% saw an improvement of at least 3 points on the scale from baseline, including 71% among patients 2-11 years old and 42% aged 12-25 years. While Part 1 of the SUNFISH study was not designed or powered to detect efficacy, the change from baseline in total MFM32 score is the primary efficacy endpoint in the ongoing Part 2 (n=180) of the trial.
Roche is planning to include the new data presented at the AAN Annual Meeting in regulatory filings with the U.S. Food and Drug Administration and European Medicines Agency during the second half of 2019.
Material provided Roche
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.
ZolgenSMA von AveXis/Novartis, sowie Risdiplam von Roche sind Medikamente die noch in der Pipeline sind. Eine neue Finanzanalyse zeigt auf, das die positive Auswirkung von Risdiplam, welch ein neuer Hoffnungsträger in der Behandlung von SMA ist, auf den Gewinn je Aktie verbindet.
Während die Erwartungen an das Mittel AVXS-101 von AveXis/Novartis möglicherweise zu hoch. Siehe hier