The US Food and Drug Administration has approved the sale of the oral drug migalastat (Galafold), manufactured by Amicus Therapeutics, for treatment of Fabry disease. The average price will be $315,000 per year for each patient, Reuters reports today (August 13).

Fabry disease is a rare genetic condition in which a type of fat called globotriaosylceramide builds up in blood vessels, the heart, the nerves, and other tissues. A mutation in a gene called alpha-galactosidase A, or GLA, leads to deficient levels of the  alpha-galactosidase A enzyme, which allows the fat to accumulate. The disease affects roughly 1 of every 40,000 to 60,000 men, though a later-onset type can affect 1 in 1,500 to 4,000 men. The disease is less common in women but can be fatal in both. Roughly 3,000 people in the US have been diagnosed with Fabry disease with more than half going without...

Until now, “treatment of Fabry disease has involved replacing the missing enzyme that causes the particular type of fat buildup in this disease,” Julie Beitz, the director of the Office of Drug Evaluation III in the Center for Drug Evaluation and Research at the FDA, says in a statement. “Galafold differs from enzyme replacement in that it increases the activity of the body’s deficient enzyme.”

Galafold is the first oral drug approved to treat Fabry disease and is the first new drug given the green light in more than 15 years. Currently, it will be used to treat patients with any one of 348 GLA variants. The drug was approved after a Phase 3 clinical trial with 60 patients showed levels of fat accumulation decreased in kidney capillaries while they took the drug. Side effects included headache, nausea, fever, and urinary tract infections.

“People with rare diseases often have limited treatment options, so research leading to new therapies is critical,” Raphael Schiffmann, director of the Baylor Scott & White Research Institute, says in an Amicus press release. “As a physician and researcher, I treated the first patient ever to receive Galafold in a clinical trial. Today, many years later, I am pleased that more patients will have access to this precision medicine.”

See The Scientist’s May 2018 Rare Diseases issue

Amicus wants to ensure that the drug is accessible to all qualifying patients. “If we’re going to have more medicines for people living with rare diseases, we’ve got to bend the cost curve,” Amicus Chief Executive John Crowley tells Reuters.

In Europe, Galafold costs between $200,000 and $250,000 each year. The $315,000 price tag in the US is set to rival the cost of Fabrazyme, which costs between $290,000 and $340,000. Any revenue made on Galafold will be put toward generating new Fabry disease treatments, Reuters reports. 

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