|
 Zuercher Kantonalbank said the outcome, in infants and toddlers
between one month and seven months with the severest form of the
genetic muscle-wasting disease, virtually guarantees risdiplam's
approval this year.
U.S. regulators are due to decide by May 24.
Roche's study of 41 patients measured whether infants could sit
independently for at least 5 seconds after 12 months of treatment.
Roche, whose risdiplam aims to join Biogen's Spinraza and Novartis's
gene therapy Zolgensma as approved SMA treatments, said no safety
problems emerged that led patients to halt treatment.
"This large, global trial confirms the efficacy of risdiplam in an
advanced and difficult-to-treat population, including many infants
whose disease had already progressed significantly before starting
treatment," said Levi Garraway, Roche's chief medical officer.
[to top of second column] |
SMA is rare, but medicines to treat it are among the industry's
costliest, creating a potentially lucrative opportunity. Zolgensma
runs $2.1 million per patient, to date the costliest one-time
treatment, while Spinraza, injected in the spine, costs $750,000 for
the first year and $375,000 annually thereafter at its U.S. list
price.
"We're almost certain of its approval," Michael Nawrath, a Zuercher
Kantonalbank analyst, said in a note to investors, predicting that
risdiplam could become the standard treatment for patients with
less-acute forms of SMA that emerge months after birth. "The sales
peak is around 2 billion Swiss francs ($2.07 billion)."
(Reporting by John Miller; Editing by Michael Shields)
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