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The Swiss drug manufacturer Novartis has received approval from the United States for its Zolgensma gene therapy against vertebral muscle atrophy – its price is $ 2.125 million.
The Food and Drug Administration on Friday approved Zolgensma for children under two years old with ADS, including those with no symptoms yet. Approval concerns babies with the most lethal form of the hereditary disease as well as those with types where debilitating symptoms may occur later.
ADS is the leading genetic cause of death in infants. The disease often causes paralysis, breathing difficulties and death in a few months for babies born with the most severe Type 1 form. ADM affects about one in 10,000 live births, with 50% to 70% of them being type 1.
"It's potentially a new standard of care for babies with the most severe form of ADS," said Dr. Emmanuelle Tiongson, pediatric neurologist in Los Angeles who provided Zolgensma to patients as part of an expanded access program.
Novartis executives defended the award, saying that a single treatment was more valuable than expensive long-term treatments, which cost hundreds of thousands of dollars a year.
The therapy uses a virus to provide a normal copy of the SMN1 gene to babies born with a defective gene and is administered by infusion.
An April review by the Independent Institute for Clinical and Economic Review (Icer) concluded that the previous estimate of $ 5 million per patient for Zolgensma from Novartis was excessive.
But Friday, Icer said that based on additional clinical data from Novartis, the FDA wide label and its introductory price, he was confident that the drug was in the upper end of its range in terms of cost-effectiveness. efficiency.
Novartis has reportedly treated up to 150 patients with Zolgensma to date. Its managing director, Vas Narasimhan, described Zolgensma as a near cure for the SMA if it was delivered shortly after birth. But the data proving its durability extend only about five years.
Novartis is awaiting European and Japanese approval later this year. Zolgensma will compete with Biogen Spinraza, the first approved treatment for ADS.
Spinraza, approved late 2016, requires infusion into the spinal canal every four months. His catalog price of $ 750,000 for the first year and $ 375,000 a year thereafter was also deemed excessive by Icer.
Wall Street analysts predict sales of $ 2 billion to Zolgensma by 2022, according to a Refinitiv poll. Spinraza sales reached $ 1.7 billion last year and are expected to reach $ 2.2 billion in 2022.
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