Roche drug for spinal muscular atrophy hits target in late-stage trial

ZURICH (Reuters) – Roche’s bid to rival Biogen and Novartis in treating spinal muscular atrophy (SMA) got a lift on Monday when the Swiss drugmaker said its drug risdiplam improved motor function in a key study.

Roche, which plans to seek approval for the investigational medicine this year, said its SUNFISH trial demonstrated statistically significant improvements in patients aged 2-25 years with Type 2 or 3 SMA.

Specifically, the study met its primary endpoint of change from baseline in the Motor Function Measure 32 (MFM-32) scale after one year of treatment with risdiplam, compared to placebo, the company said.

Risdiplam is a survival motor neuron-2 (SMN2) splicing modifier, designed to increase SMN protein levels in the central nervous system and peripheral tissues.

Spinal muscular atrophy, which hits 1 in every 10-11,000 newborns, has been a target of significant interest as drugmakers move into a rare disease area that commands ultra-high prices. Biogen’s Spinraza, approved in 2016, lists at $750,000 in year one and $375,000 thereafter, while Novartis’s Zolgensma, approved for use in babies and toddlers, is the costliest one-time treatment at $2.1 million.

Roche’s risdiplam, which would be the third treatment to market, is taken by mouth, as opposed to injected into the spine like Spinraza. Roche is also hoping data from its studies stacks up to that of Zolgensma, including in older SMA patients with forms of the disease that may emerge months after their birth but leave them with profound disabilities.

In its statement, Roche underscored the safety profile of risdiplam just weeks after Novartis’s trial of Zolgensma in a similar population of SMA patients was halted by the U.S. Food and Drug Administration when primate study raised concerns of possible nerve damage or loss.

“No treatment related safety findings leading to study withdrawal have been seen in any risdiplam trial to date,” Roche said, adding it would release specific data from the SUNFISH trial at an upcoming medical conference.

Roche has partnered on risdiplam with PTC Therapeutics.