Roche presents data from the risdiplam pivotal FIREFISH & SUNFISH studies in SMA Typ I-III at the 2019 AAN

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.[3] 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.[3] 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

Publicerat av

Liz

Att acceptera vad som händer och låt trollen i ljuset, kanske du borde att tro mer på dig själv

En kommentar till “Roche presents data from the risdiplam pivotal FIREFISH & SUNFISH studies in SMA Typ I-III at the 2019 AAN”

  1. Great results.👍🙏
    The side effects are the usual and not dramatic. Impressive I think that none of the children need a trach. Really promising, even for adults with SMA. 👍 Thank U Roche. 🙏

Kommentarer inaktiverade.