FDA Approves World’s Most Expensive Treatment

The Food and Drug Administration has approved a new gene therapy for a rare muscle-wasting disease that is priced at $2.1 million, believed to be highest ever for a one-time treatment. The treatment, to be sold as Zolgensma, is the property of Swiss drugmaker Novartis (NYSE: NVS). The company plans to launch the drug immediately, and expects roughly 1,100 patients to be eligible for it at launch.

Zolgensma is a one-time therapy that works to treat spinal muscular atrophy, a rare genetic condition that affects the motor nerve cells in the spinal cord, at the genetic level. Babies born with the condition have impaired reflexes and lessened muscular and head control. Children with a severe form of the disorder often die before they turn 2.

According to the SMA Foundation, spinal muscular atrophy affects an estimated 10,000 to 25,000 individuals in the U.S. Roughly 1 in 11,000 babies are born with the condition. Until 2016, there were no treatments available for the condition. Zolgensma is now approved for children under 2 with all forms of spinal muscular atrophy.

At $2.1 million a treatment, Zolgensma will be too pricey for most American families without the approval of insurance companies. Novartis will allow insurers to pay for the treatment over five years, which would average out to $425,000 per year. The payment plans will be administered by Accredo, the specialty pharmacy unit of Express Scripts now owned by Cigna. Novartis also said it would provide a discount if a patient who takes Zolgensma dies or eventually needs permanent ventilator support. The amount of that discount has not yet been disclosed.

Novartis says that the price of Zolgensma is comparable to the prices for other treatments of ultrarare disorders. Spinraza, another drug approved to treat spinal muscular atrophy, costs $750,000 for the first year of treatment, then about $375,000 a year after that, totaling about $4 million for a decade of treatment. Zolgensma is a single-use treatment.