(Reuters) – U.S. health regulators on Friday approved Amicus Therapeutics’ Galafold, the first oral therapy to treat Fabry disease, a rare, sometimes fatal condition in which accumulation of fat damages several organs.
Galafold, known chemically as migalastat, will be the first new Fabry treatment on the U.S. market in over 15 years, and will compete with Sanofi SA’s infused Fabrazyme.
The drug is already approved in seven other markets, including Europe, Japan, Australia and Canada.
Galafold is a long-term treatment for adult patients with specific genetic mutations. That accounts for about 35 percent to 50 percent of the 10,000 diagnosed Fabry patients worldwide. An estimated 3,000 people in the United States are diagnosed with the disease.
Reporting by Robin Respaut; editing by Phil Berlowitz, Bill Berkrot, Michele Gershberg and Jonathan Oatis