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 treatment.
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