FDA approves $2M medicine, most expensive ever

U.S. regulators have approved the most expensive medicine ever, for a rare disorder destroying a baby's muscle control and killing nearly all of those with the most common type of the disease within a couple of years.

The treatment is priced at $2.125 million. Out-of-pocket costs for patients will vary based on insurance coverage.

The medicine, sold by the Swiss drugmaker Novartis, is a gene therapy treating an inherited condition called spinal muscular atrophy. The treatment targets a defective gene weakening a child's muscles so dramatically they become unable to move, and eventually unable to swallow or breathe. It strikes about 400 babies born in the U.S. each year.

The Food and Drug Administration on Friday approved the treatment, called Zolgensma, for all children younger than 2 who are confirmed by a genetic test to have any of the four types of the disease. The therapy is a one-time infusion.

Novartis said it will let insurers make payments over five years, at $425,000 per year, and will give partial rebates if the treatment doesn't work.

The one other medicine for the disease approved in the U.S. is a drug called Spinraza. Instead of a one-time treatment, it must be given every four months. Biogen, Spinraza's maker, charges a list price of $750,000 for the first year and then $350,000 per year after that.

The independent not-for-profit group Institute for Clinical and Economic Review, which rates the value of expensive new medicines, calculated the price of the new gene therapy is justifiable at a cost of $1.2 million to $2.1 million because it "dramatically transforms the lives of families affected by this devastating disease."

ICER's president, Dr. Steven D. Pearson, called the treatment's price "a positive outcome for patients and the entire health system."

The defective gene causing spinal muscular atrophy prevents the body from making enough of a protein allowing nerves controlling movement to work normally. The nerves die off without the protein.

In the most common type, which is also the most severe, at least 90% of patients die by age 2, and any still alive need a ventilator to breathe. Children with less-severe types become disabled more slowly and can live for up to a couple decades.