Roche has presented one-year data from its pivotal Part II of the SUNFISH trial, evaluating risdiplam in certain people with spinal muscular atrophy (SMA).
The pharma giant revealed that in patients aged 2-25 years with type 2 or 3 SMA, the treatment showed significant improvement in motor function. The change from baseline in the primary endpoint of the Motor Function Measure scale (MFM-32) was significantly greater in people treated with the drug than those with placebo.
Roche also announced that the secondary endpoint of Revised Upper Limb Module also showed improvement, with a 1.59 point difference.
An exploratory subgroup analyses showed that the strongest responses in MFM-32 versus placebo were observed in the youngest age group of two to five years old, as 78.1% of the patients achieved ≥3 point increase, compared to 52.9% with placebo.
Risdiplam is the “first potential treatment to have pivotal placebo-controlled data in a broad population of patients, including children, teenagers and adults,” said SUNFISH principal investigator Eugenio Mercuri, department of Paediatric Neurology, Catholic University.
He continued, “The data suggest that risdiplam can preserve and potentially enable greater independence through improved motor function in people with Type 2 or non-ambulant Type 3 SMA.”
Further, the SUNFISH study showed safety profiles consistent with the drug’s known profiles.
In November 2019, the US Food and Drug Administration granted Priority Review for the treatment with a decision for approval by May 24, 2020.