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The Novartis logo is visible in his agency in Schweizerhalle, near Basel, Switzerland. Reuters
The Swiss drug manufacturer Novartis has obtained approval from the United States for its Zolgensma gene therapy against spinal muscular atrophy (SMA), the leading genetic cause of death in infants, and has estimated the unique treatment to $ 2.125 million.
The Food and Drug Administration has approved Zolgensma for children under two years of age with ADS, including those who do not have 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.
"This is potentially a new standard of care for infants with the most severe form of ADS," said Dr. Emmanuelle Tiongson, pediatric neurologist at Children's Hospital in Los Angeles, who provided Zolgensma to patients in part of an expanded access program. "The job now is to negotiate with insurers to make it a long-term economy."
Novartis executives defended the award, saying that a single treatment was more valuable than an expensive long-term treatment costing several hundred thousand dollars a year.
Novartis launched a debate on the value of gene therapy last year, estimating that its treatment would be profitable at $ 5 million per patient. In April, an independent US group, the Institute for Economic and Economic Review (ICER), concluded that Novartis' estimate of value for Zolgensma was excessive.
However, on Friday, ICER stated that based on additional clinical data from Novartis, the FDA wide label and its introductory price, ICER believed that the drug was within the upper end of its range in terms of cost-effectiveness. efficiency.
Novartis indicated that it offers health insurers the possibility of making installment payments for Zolgensma, as well as refunds if the treatment was not working and initial discounts for payers committing to standardized coverage conditions.
Novartis chief executive Vas Narasimhan has a lot to say about Zolgensma, which he describes as a virtual cure for the SMA if it is delivered shortly after birth. But the data proving its durability extend only about five years. The therapy uses a virus to provide a normal copy of the SMN1 gene to babies born with a defective gene. It is delivered by infusion.
Novartis is awaiting European and Japanese approval later this year. Zolgensma will compete with Spinraza of Biogen Inc., the first approved treatment for ADM.
The disease often causes paralysis, breathing difficulties and death in a few months for infants born with the most severe type I form. SMA affects about one in 10,000 live births, 50% to 70% of children are born with the disease. between them being affected by a type I disease. Spinraza, approved late 2016, requires infusion into the spinal cbad every four months. Its catalog price of $ 750,000 for the first year and $ 375,000 thereafter was also deemed excessive by ICER.
Some neurologists consider that gene therapy is becoming the treatment of choice for newborns with severe AS, while recognizing that families may choose to wait for the data relating to the condition. safety and long-term effectiveness of Zolgensma. Novartis is investigating whether the death of a critically ill baby being treated with Zolgensma was treatment-related.
"Most families will want to do gene therapy because it avoids frequent spinal taps," said Dr. Russell Butterfield of the University of Utah in Salt Lake City. Butterfield has received payments from Biogen for consultation.
The FDA has approved Zolgensma on the basis of clinical trials of 36 patients aged 2 weeks to 8 months. According to the agency, patients treated with Zolgensma showed a significant improvement in developmental milestones such as head control and the ability to sit down.
The most common side effects of Zolgensma are elevated liver enzymes and vomiting. The FDA requires that the Zolgensma label contain a warning that severe liver damage may occur. With additional studies underway, Novartis said it has so far treated more than 150 patients with Zolgensma, acquired when AveXis was purchased for $ 8.7 billion last year.
Wall Street badysts expect a turnover of $ 2 billion by 2022, according to a survey of Refinitiv. Spinraza sales reached $ 1.7 billion last year and are expected to reach $ 2.2 billion in 2022. Roche is developing the oral drug risdiplam and is considering approving it later this year .
Agencies
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