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Amicus Therapeutics prices rare disease drug below rival's

Cranbury, N.J.-based Amicus Therapeutics Inc. said its recently approved Fabry disease treatment, Galafold, would cost, on average, $315,000 per year in the U.S.

The drug received approval from the U.S. Food and Drug Administration last week and is the first new therapy approved in the U.S. to treat the inherited disorder in more than 15 years.

The therapy will be cheaper than Sanofi's Fabrazyme, the current standard of care, which Amicus estimated had an average annual list price of $344,000.

Fabrazyme was approved by the U.S. Food and Drug Administration in April 2003 and had 2017 global sales of €722 million.

Galafold, which generated $36.9 million in revenues during 2017, is already approved in the U.K., Canada, Japan and the EU.

Fabry disease results from the buildup of a particular type of fat in the blood vessels, kidneys, heart, nerves and other organs. Patients with the disease develop slowly progressive kidney disease, enlargement of the heart, abnormal heart rhythm, stroke and early death.

In the U.S., it is estimated that more than 3,000 people are living with Fabry disease, and more than an estimated 50% of these diagnosed patients are untreated.