Swiss drugmaker Novartis on Friday won U.S. approval for its gene therapy Zolgensma for spinal muscular atrophy (SMA), the leading genetic cause of death in infants, and priced the one-time treatment at a record $2.125 million.
The Food and Drug Administration approved Zolgensma for children under the age of two with SMA, including those not yet showing symptoms. The approval covers babies with the deadliest form of the inherited disease as well as those with types where debilitating symptoms may set in later.
“This is potentially a new standard of care for babies with the most serious form of SMA,” said Dr. Emmanuelle Tiongson, a pediatric neurologist at Children’s Hospital Los Angeles who has provided Zolgensma to patients under an expanded access program. “The job now is trying to negotiate with insurers that this would be a long-term savings.”
Novartis executives defended the price, saying that a one-time treatment is more valuable than expensive long-term treatments that cost several hundred thousand dollars a year.
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Novartis touched off a debate over what gene therapy is worth last year, estimating its treatment would be cost-effective at up to $5 million per patient. A review in April by an independent U.S. group, the Institute for Clinical and Economic Review (ICER), concluded Novartis’ value estimate for Zolgensma was excessive.
But on Friday, ICER said that based on Novartis’ additional clinical data, the broad FDA label and its launch price, it believed that the drug fell within the upper bound of its range for cost-effectiveness.
Novartis said it was offering health insurers the option of installment payments for Zolgensma as well as refunds if the treatment does not work and upfront discounts for payers who commit to standardized coverage terms.
Novartis Chief Executive Vas Narasimhan has much riding on Zolgensma, describing it as a near cure for SMA if delivered soon after birth. But data proving its durability extends to 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.