FDA Approves the Priciest Ever Medicine That Treats Rare Genetic Disorder

Zolgensma is for all children under two years of age with a deadly genetic disease, which costs more than $2 million.

FDA Approves Priciest Medicine Treats Genetic Disorder

The U.S. Food and Drug Administration (FDA) has approved the most expensive medication ever for a rare genetic disorder, which destroys a baby’s muscle control and causes death within two years. The drug is priced at $2.125 million; however, out-of-pocket costs for patients may vary based on their insurance coverage.

On Friday, the FDA approved the drug called Zolgensma for all children below two years of age, who are diagnosed by a genetic test to have any of the three types of the disorder. Zolgensma therapy includes a one-time infusion, which takes approximately an hour.

Swiss drug-maker Novartis sells the drug, which is a gene therapy that treats a genetic disorder known as spinal muscular atrophy. The drug targets a defective gene that destroys a child’s muscles to an extent where they are unable to move, and unable to even swallow or breathe. In the United States, this condition affects nearly 400 babies each year.

The drug manufacturer said it would let insurers make payments over a period of five years ($425,000 per year). It also said it would give partial rebates if the drug fails to work.

Another drug for the same condition approved by the FDA in the U.S. is Spinraza. This drug includes a therapy that must be given every four months. Spinraza’s maker Biogen charges a list price of $750,000 for the first year and $350,000 per year after that.

According to the independent nonprofit Institute for Clinical and Economic Review (ICER) that rates the value of expensive new drugs, the new gene therapy costs $1.2 million to $2.1 million because it “dramatically transforms the lives of families affected by this devastating disease.”

The president of ICER Dr. Steven D. Pearson said, “The treatment’s price is a positive outcome for patients and the entire health system.”

In spinal muscular atrophy, the defective gene does not allow the body from making enough protein that nourishes the nerves to control movement, leading to the nerve cell death.

Zolgensma supplies a healthy copy of that faulty gene, allowing nerve cells to start producing the much-needed protein. This eventually halts the deterioration of the nerve cells, allowing the child to develop normally.

According to clinical studies, babies with the severe form of the genetic disorder who got Zolgensma within six months of birth experienced limited muscle problems and those who got the gene therapy earliest did great.

Early diagnosis is important, so Novartis is working with states to get genetic testing for infants required at birth. The drug-manufacturer expects most states will be meeting that requirement by next year. The FDA said that the side effects Zolgensma included vomiting and potential liver damage; therefore, patients should be monitored for the first few months after introducing the drug.