The Food and Drug Administration has approved Novartis’ $2.1 million gene therapy for spinal muscular atrophy — making it the world’s most expensive drug.  The therapy, Zolgensma, is a one-time treatment for spinal muscular atrophy, a muscle-wasting disease and leading genetic cause of infant mortality that affects one in every 11,000 births. Novartis had previously said it could price the treatment between $1.5 million to $5 million.

Novartis said the treatment will cost $2.1 million — or $425,000 a year spread out over five years. The company said it’s “working closely with insurers to create 5-year agreements based on success of the treatment as well as other novel pay-over-time options.” It’s currently in “advanced discussions” with more than 15 insurers on payment options.

Zolgensma works by providing a functional copy of the defective gene responsible for SMA to halt the disease’s progression via a one-time intravenous infusion. The US Food and Drug Administration said the drug’s safety had been tested in an ongoing clinical trial and a completed clinical trial involving 36 patients between the ages of two weeks and eight months.

The most common side effects of Zolgensma are elevated liver enzymes and vomiting, the FDA said. Gene and cell therapies leverage the biology to reverse diseases ranging from congenital blindness to pediatric leukemia.

Food and Drug Administration

• The Food and Drug Administration (FDA or USFDA) is a federal agency of the United States Department of Health and Human Services, one of the United States federal executive departments.
• The FDA is responsible for protecting and promoting public health through the control and supervision of food safety, tobacco products, dietary supplements, prescription and over-the-counter pharmaceutical drugs (medications), vaccines, biopharmaceuticals, blood transfusions, medical devices, electromagnetic radiation emitting devices (ERED), cosmetics, animal foods & feed and veterinary products.

Gene Therapy

• Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein.
• If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein.
• A gene that is inserted directly into a cell usually does not function. Instead, a carrier called a vector is genetically engineered to deliver the gene.
• Certain viruses are often used as vectors because they can deliver the new gene by infecting the cell. The viruses are modified so they can’t cause disease when used in people.