SMA is an inherited, progressive neuromuscular disorder characterised by muscle atrophy and other complications. Credit: Darwin Laganzon from Pixabay.

Roche has reported one-year data from the second part of the SUNFISH clinical trial conducted to assess risdiplam in type 2 or 3 spinal muscular atrophy (SMA) patients aged two to 25 years.

Data revealed that significantly more patients treated with the drug experienced a change in the Motor Function Measure scale (MFM-32) from baseline, the primary endpoint, compared to placebo.

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Improvement was also observed on a key secondary endpoint of the Revised Upper Limb Module (RULM).

Risdiplam is an investigational, oral survival motor neuron-2 (SMN2) splicing modifier. It is being developed in alliance with the SMA Foundation and PTC Therapeutics.

SUNFISH is a two-part, double-blind pivotal study where the first part identified the dose for the confirmatory second part. Part two used MFM-32 to assess motor function at 12 months.

Exploratory subgroup analyses found the strongest MFM-32 responses in the youngest age group of two to five years. Meanwhile, disease stabilisation was achieved in the 18-25 age group.

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The safety profile of Roche’s drug was found to be consistent with its known profile, with no new safety signals. The most common adverse events were upper respiratory tract infection, nasopharyngitis, pyrexia, headache, diarrhoea, vomiting and cough.

Roche chief medical officer and Global Product Development head Levi Garraway said: “We are very encouraged by the positive results in this broad group of SMA patients, many of whom are under-served and under-represented in clinical trials.

“This study has helped us understand which measurement scales are the most relevant for patients, as well as the importance of stabilisation in people with more established disease.”

Earlier this month, the company reported positive top-line data of risdiplam from part two of the open-label FIREFISH study conducted in babies with type 1 SMA.