Roche has announced data from its SUNFISH trial, evaluating risdiplam (RG7916) in people aged two to 25 years with type II or III spinal muscular atrophy (SMA).
The company says the drug met its primary endpoint, defined as change from baseline in the Motor Function Measure 32 (MFM-32) scale after one year of treatment, compared to placebo.
The pharma giant also confirmed that safety data for the treatment was consistent with its known safety profile, and that no new issues were identified.
The drug in question is an investigational survival motor neuron-2 (SMN2) splicing modifier, designed to durably increase and sustain SMN protein levels both throughout the central nervous system and peripheral tissues of the body. It is currently being studied in a broad clinical trial programme in SMA, with patients ranging from birth to 60 years old, and includes patients previously treated with SMA-targeting therapies.
“The positive outcome of this trial is an important milestone for people with type II or III SMA, too many of whom remain untreated,” said Levi Garraway, Roche’s chief medical officer and head of global product development.
He continued to say that the trial is the “largest placebo-controlled study ever undertaken in Type I or III SMA patients,” before thanking the community for their “partnership and look forward to sharing these results with regulators and bringing risdiplam to people living with this condition.”
Spinal muscular atrophy (SMA) is a severe, inherited, progressive neuromuscular disease that causes devastating muscle atrophy and disease-related complications. It is the most common genetic cause of infant mortality and one of the most common rare diseases, affecting approximately one in 11,000 babies.