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TThe Food and Drug Administration approved Friday the first gene therapy against a type of spinal muscular atrophy, a vital treatment for infants, which will also be the most expensive drug in the world.
Known as Zolgensma, this gene therapy treats children under 2 years of age with spinal muscular atrophy, a hereditary neuromuscular disease leading to a progressive loss of muscle function. The most severe form of SMA causes infant death or permanent respiratory support at the age of 2 years. The disease is caused by a defect in a gene that makes the SMN, a protein necessary for the survival of motoneurons. Zolgensma uses a redesigned virus to deliver a functional copy of the defective gene so that the SMN protein can be produced.
Novartis estimates Zolgensma at $ 2.125 million, an annualized cost of $ 425,000 a year for five years, the company said.
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The launch of Zolgensma will be a big test for Novartis and its CEO, Vas Narasimhan, now two years at work. Shareholders expect gene therapy to generate exceptional sales and justify the $ 8.7 billion spent by Novartis to acquire it last year.
To be commercially successful, Novartis must convince physicians who treat AMS patients that the benefits of a single injection of Zolgensma for muscle preservation will be sustainable. The complex arrangements for payment and reimbursement of insurance required for expensive gene therapies must be handled with cunning.
Novartis is likely to face negative reactions from critics who believe that charging drugs for drugs, no matter how effective, makes it unaffordable for a healthcare system that is already in financial difficulty.
There is also competition. Spinraza, approved late 2016 and sold by Biogen, has already been used to successfully treat thousands of patients with severe and mild forms of ADS. The drug requires regular injections into the spine costing $ 750,000 in the first year and $ 375,000 thereafter, for life. Last year, sales totaled $ 1.7 billion. Zolgensma may be more practical than Spinraza, but Roche is currently developing a daily SMA pill, called risdiplam, which could hit the market in 2020.
The FDA has approved Zolgensma for treating children under 2 years of age with ADS, regardless of their genetic mutation. In a pivotal clinical trial and an ongoing clinical trial, a majority of infants and young children who received an injection of Zolgensma remained alive, could breathe on their own, and had improvements in motor milestones, such as being able to survive. Sit without support.
Zolgensma "is significantly better than any other treatment, especially in type 1 clinical trials that we have published," Narasimhan told STAT in a recent interview. "It's clear that parents will know right away that this is a drug that works extremely well in these infants and has this kind of impact on their well-being."
In his announcement, FDA Acting Commissioner Ned Sharpless said the approval marked "a new step in the transformative power of gene and cell therapies to treat a wide range of diseases."
A study conducted by Jefferies analysts of 30 physicians currently treating patients with ADS revealed that 30% of them would use Zolgensma in their newly diagnosed patients one year after launch. Doctors were also interested in combinations of Zolgensma and Spinraza. Jefferies expects Zolgensma sales to reach $ 2.6 billion, more than the $ 1.9 million expected by consensus.
Biogen does not agree, this is not surprising. In a recent teleconference, leaders said that even with the arrival of Zolgensma, Spinraza remained the standard of care for the treatment of ADS, based on the broader label of the drug and more than 7,000 treated patients, some for six years. Spinraza is approved for all types of ADS – from the sickest type 1 infants to adults with benign forms of the disease, when the loss of muscle function begins later and is more progressive.
"Biogen wants new therapeutic options to help people with this rare disease," said the company in a statement released after Zolgensma's approval, "We are proud to have helped more than 7,500 people with ADS." Spinraza remains the only treatment available for a large group of AS patients. "
Dr. John Brandsema, pediatric neurologist at Philadelphia Children's Hospital, treated patients with AMS with Spinraza and Zolgensma. He thinks the comparisons are premature.
"The academic community is reluctant to directly compare patients with the gene transfer trial. [Zolgensma] to nusinersen patients [Spinraza] trial. They were different in their inclusion criteria, their age at the start of treatment and some of the outcome measures at the study, "Brandsema told STAT. "I think you really need real experience to be able to make comparisons at this level.We have not yet had any real experience in gene transfer." Brandsema has received consulting fees from Biogen and AveXis, the biotechnology company that developed Zolgensma and was acquired by Novartis.
Dr. Ed Smith, an ADM expert from Duke University, said his patients and their caregivers were already sensitized to Zolgensma.
"I would say that to talk to patients and families who are currently practicing Spinraza, many of them are eagerly awaiting the option of a single dose of gene therapy if it is an option for them," he said. Smith told STAT. "Most of them I asked, and I asked most of them, are looking forward to this option and want to know if it will be an option for me."
About that $ 2.1 million Zolgensma Label Price: Novartis defends its pricing decision, calling the treatment "very cost-effective," "fair and reasonable" given the benefits demonstrated in clinical trials. Novartis pointed out that chronic injections of Spinraza cost more than $ 4 million over five years.
Novartis said it was working with insurers to implement long-term and outcome-based payment agreements to speed up patient access and Zolgensma reimbursement.
Significantly, Novartis will not be rejected afterwards. Institute for Clinical and Economic Review, a non-profit organization that has become an unofficial force in the country to evaluate the economic benefits of new drugs. ICER on Friday approved Novartis' pricing strategy. An updated ICER cost-effectiveness analysis revealed that Zolgensma, at $ 2.1 million, was only slightly higher than its value-based benchmark. A previous ICER analysis had established the justifiable cost of Zolgensma at $ 1.5 million.
"Zolgensma is radically transforming the lives of families affected by this devastating disease and, given the new efficacy data available for the pre-symptomatic population, the price announced today is in the upper limit of the range. price reference based on the value of ICER, "said Dr. ICER. Steven D. Pearson, President of ICER.
Peter Bach, director of the Center for Health Policy and Results at the Memorial Sloan Kettering Cancer Center, is concerned about the price of Zolgensma and believes that the recent cost-effectiveness analysis of ICER takes too many liberties.
"You can look at it in two ways. It's an amazing treatment and only a few kids will need it. One million here, and one million there, is not worth more than the shoulder, "he said. "Or we have a big problem. Biopharma has been completely reoriented towards rare diseases because the market will tolerate all prices and the FDA will require fairly minimal data. "
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