A newly approved gene therapy to treat spinal muscular atrophy is the priciest yet, with a list price of $2.1 million, the Associated Press reports.
The US Food and Drug Administration approved Novartis's Zolgensma Friday for children under the age of two who are shown by genetic testing to have one of three forms of the disease, the AP adds. The Guardian notes that SMA causes paralysis, breathing difficulties and death, and is the leading genetic cause of death among infants.
According to Novartis, Zolgensma is a one-time treatment that delivers a working copy of the SMN gene to prevent disease progression.
The AP reports that that the Institute for Clinical and Economic Review has determined that a cost of between $1.2 million to $2.1 million is justifiable for the treatment as it "dramatically transforms the lives of families affected by this devastating disease." ICER had, the Guardian notes, called drug maker's previous $5 million price tag estimate too high. It also says that Novartis has defended the drug's current cost, arguing that it is more valuable than other expensive, long-term treatments.
The AP adds that health insurers may make installment payments over five years and could get partial rebates based on how well the treatment works, but that the cost to patients will vary based on their insurance coverage.