Swiss drugmaker Novartis won U.S. approval for its gene therapy Zolgensma for spinal muscular atrophy (SMA) and priced the one-time treatment at a record $2.125 million
ZURICH/NEW YORK - Swiss drugmaker Novartis on Friday 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.
Novartis is expecting European and Japanese approval later this year. Zolgensma will compete with Biogen Inc’s Spinraza, the first approved treatment for SMA. “Most families will want to do the gene therapy since it avoids the frequent spinal taps,” said Dr. Russell Butterfield of the University of Utah in Salt Lake City. Butterfield has received payments from Biogen for consulting.
“Babies are losing motor neurons from the day they are born, so the ability to treat them as early as possible is the way you get maximum value out of the therapy,” David Lennon, who heads Novartis’ AveXis unit, said in a recent interview.
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