|
 U.S. regulators are expected to make a decision this month on
whether to approve Zolgensma, a one-time therapy seen as a potential
long-term solution for the rare disease that is the leading genetic
cause of death in infants. The company has previously said it could
price the treatment in a range of $1.5 million to $5 million.
“We won’t be announcing the price until we get the approval. But our
overall goal is to be at a fraction of what is the current standard
of care, and the current standard of care for treating these
patients is $4 million to $5 million over 10 years,” Novartis Chief
Executive Vas Narasimhan told reporters on Wednesday in Boston.
The current treatment for SMA, which can lead to paralysis,
breathing difficulty and early death, is Biogen Inc's Spinraza. That
drug has a list price of $750,000 for the initial year and $375,000
annually thereafter.
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Narasimhan said his company's ultra-rare disease therapy would be
cost effective in a range of $4.6 million to $5.4 million. “We want
to be far lower than that,” he said.
Novartis is expecting the drug initially to be approved for infants
and is in discussions with the U.S. Food and Drug Administration
about whether approval will include other forms of the disease
beyond the most serious Type 1, he said.
The company is also in late stage discussions with commercial
insurers over the price of the treatment.
(Reporting by Caroline Humer; Editing by Bill Berkrot)
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