Therapy Areas: Central Nervous System
Roche meets primary endpoint in part two Risdiplam SUNFISH trial
13 November 2019 -

Switzerland-based Roche has met its primary endpoint in part two of the SUNFISH trial evaluating Risdiplam intended for the treatment of Type 2 or 3 spinal muscular atrophy in people aged two to 25 years, it was reported yesterday.

The product has been designed to be a survival motor neuron-2 splicing modifier. It works by durably increasing and maintaining survival motor neuron protein levels throughout the central nervous system and also in the peripheral tissues of the body. The primary endpoint met in part 2 of the trail is change from baseline in the Motor Function Measure 32 (MFM-32) scale following a year of treatment with Risdiplam, in comparison to placebo.

The company stated that safety for the investigational drug was on par with its known safety profile with no new safety signals detected.

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