Swiss pharmaceutical giant Novartis has won approval for a new gene therapy drug which is priced at a record £1.67m per treatment.
Named Zolgensma, the drug has been approved by US regulators for treating babies with spinal muscular atrophy (SMA) – a rare and often fatal disease.
The new one-time treatment uses a virus to replace the defective gene which causes SMA with a copy that functions properly.
The regulatory approval allows Zolgensma to be used on babies with the deadliest form of the disease as well as ones whose lives could be impacted later.
Babies affected by the most severe form of the progressive condition rarely survive beyond two years of age.
“This is potentially a new standard of care for babies with the most serious form of SMA,” pediatric neurologist Dr Emmanuelle Tiongson said.
“The job now is trying to negotiate with insurers that this would be a long-term savings,” added Dr Tiongson, who provided Zolgensma to patients under an expanded access program.
Executives at the pharmaceutical giant defended their price of the drug and claimed that the one-time treatment, which is delivered via spinal infusion, is more valuable than alternatives which cost hundreds of thousands of dollars a year.
The cost of gene therapy drugs has been a subject of hot debate. An independent review in April by the Institute for Clinical and Economic Review (ICER) in the US claimed that Novartis was overestimating the value of Zolgensma.
But following the approval on Friday, the ICER retracted this claim, saying that based on additional clinical data provided by Novartis it now believed that Zolgensma was, while still expensive, within range for being cost-effective.
Novartis has also submitted the drug for review to the European Medicines Agency and a decision from the regulator is expected in the coming months.