|
 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, compared to a placebo.
The muscle-wasting disease, 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 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.
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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.
(Reporting by John Miller, editing by John Revill)
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