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Roche's risdiplam hits main goal in spinal muscular atrophy type 2 or 3

Published on 11/11/19 at 12:15pm

Roche has lifted the curtain on new data which shows that its investigational survival motor neuron-2 (SMN2) splicing modifier risdiplam met its primary endpoint in the treatment of type 2 or 3 spinal muscular atrophy (SMA) in patients between the age of 2 and 25.

The new findings confirmed that the therapy generated a significant change from baseline on the Motor Function Measure 32 (MFM-32) scale after 12 months of treatment compared to placebo, smashing the main goal of the study.

The company also confirmed that the therapy’s safety profile was found to be consistent with previous data and no trial participants withdrew as a result of such concerns.

“The positive outcome of this trial is an important milestone for people with Type 2 or 3 SMA, too many of whom remain untreated,” explained Dr Levi Garraway, Roche’s Chief Medical Officer and Head of Global Product Development. “SUNFISH is the largest placebo-controlled study ever undertaken in Type 2 or 3 SMA patients. We thank the SMA community for their partnership and look forward to sharing these results with regulators and bringing risdiplam to people living with this condition.”

Matt Fellows

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