Gene therapy gets FDA approval – and a $ 2 million price

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And then comes the price: $ 2.125 million for one-time treatment.

Gene therapy, called Zolgensma, will be marketed by AveXis, whose parent company is Novartis.

"Today's approval marks a new step in the transformative power of gene and cell therapies to treat a wide range of diseases," said Friday Dr. Ned Sharpless, Acting Commissioner of Health. the FDA, in a statement. "The potential of gene therapy products to change the lives of these patients who may have faced a life-threatening illness or, worse, death, gives hope in the future."

The list price, which should be in the low millions, immediately revived the controversy around rising drug costs and how to pay for them.

"It's emblematic of our failing system that actually forces us to pay the price that pharmaceutical companies require for new life-saving drugs," said David Mitchell, founder of the Patient Advocacy Group for Affordable Drugs. in a statement.

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Novartis executives said Friday that the price had reached their profitability targets, with the newly approved treatment costing about half of what another treatment for the disorder, Spinraza, could cost more than 10 years. They stated that the company will have patient support and payment programs and that it does not expect the costs to be prohibitive for patients in need of treatment.

"We have not paid the polio vaccine on the basis of future savings for children not having to live in steel lungs," Mitchell said in criticizing the decision to pricing.

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Clinical Services Manager Cigna Corp. said in a statement that the health insurer was planning to "continue the work started with AveXis to find unique solutions, such as installments and outcome-based agreements for life-changing gene therapies"

Spinal muscular atrophy is an inherited condition resulting from a defective gene that causes the death of nerve cells responsible for the movement of the muscles that allows us to walk, talk, breathe and swallow, according to the National Institute of Disorders neurological and stroke. Zolgensma replaces a working copy of this gene in these nerve cells before they die and the development of their symptoms.

It is estimated that up to 1 in every 8,000 babies is affected by one of the genetic mutations that causes it. According to a Novartis release, this includes about 450 to 500 infants in the United States per year.

Sharpless said on Friday that the agency planned to speed up the process of reviewing products that meet unmet medical needs and market them.

In January, FDA executives said they expect the agency to approve 10 to 20 cell and gene therapy products every year by 2025.
In a CNBC editorial last week, Dr. Vas Narasimhan, Chief Executive Officer of Novartis, called for a new pricing and payment model for single-drug treatments, such as Zolgensma, as opposed to other treatments whose costs are higher. 'accumulate with time.

"Health care systems, however, are accustomed to treating chronic diseases according to a pay-as-you-go model, spreading the costs over months and years," Narasimhan said. "They are currently unprepared to pay for an upsurge in new single-treatment therapies that could potentially yield lifetime benefits."

But Mitchell, himself a cancer patient, said the company was abusing a faulty system, "forcing families to answer a question:" What are you willing to pay? to save the life of your child? ""

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