ZURICH/NEW YORK - Swiss drugmaker Novartis won U.S. approval for its gene therap...
ZURICH/NEW YORK - Swiss drugmaker Novartis won U.S. approval for its gene therapy Zolgensma for spinal muscular atrophy , the leading genetic cause of death in infants, and priced the one-time treatment at a record $2.125 million.
Novartis touched off a debate over what gene therapy is worth last year, estimating its treatment would be cost effective at up to $5 million per patient. A review in April by an independent U.S. group, the Institute for Clinical and Economic Review , concluded Novartis’ value estimate for Zolgensma was excessive.
The disease often leads to paralysis, breathing difficulty and death within months for babies born with the most serious Type I form. SMA affects about one in every 10,000 live births, with 50 percent to 70 percent having Type I disease. The FDA approval was based on a study of 15 children with Type 1 SMA, all of whom are still alive up to five years after treatment. Of 12 babies who got the now-approved dose, 11 could sit unassisted, and at least two are walking, researchers reported.Novartis acquired Zolgensma with its $8.7 billion purchase of Chicago-based AveXis last year.
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