Roche’s risdiplam hits main goal in infants with spinal muscular atrophy

pharmafile | January 23, 2020 | News story | Research and Development |  Roche, pharma, risdiplam, spinal muscular atrophy 

Roche has announced the success of its survival motor neuron-2 (SMN2) splicing modifier risdiplam in a new trial investigating its efficacy in the treatment of type 1 spinal muscular atrophy (SMA) in infants aged 1-7 months.

The manufacturer confirmed that its product met its primary endpoint of the proportion of participants sitting up without support for at least five seconds at 12-months of treatment, as measured by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III).

The safety profile for the therapy was also found to be consistent with previous data; of the more than 400 patients treated with the drug in trials so far, none have been forced to withdraw as a result of safety findings.

“This large, global trial confirms the efficacy of risdiplam in an advanced and difficult-to-treat population, including many infants whose disease had already progressed significantly before starting treatment,” commented Dr Levi Garraway, Roche’s Chief Medical Officer and Head of Global Product Development. “We are very encouraged by these results and we look forward to sharing them with regulators. We also thank the entire SMA community for their continued partnership.”

Roche confirmed that full data from the study would be presented at an upcoming medical conference.

Matt Fellows