This move by Japan’s Ministry of Health, Labour and Welfare makes Galafold, developed by Amicus, the first and only oral precision medicine approved for Fabry disease in the country.
“As a principal investigator in the Galafold pivotal studies with extensive experience treating Fabry disease, it can be said that significant unmet need remains. Galafold has a unique mechanism of action that has demonstrated compelling results in naïve and treatment-experienced Fabry patients who have amenable mutations. This differentiated treatment option is good news for the many Fabry patients in Japan who have an amenable mutation,” Toya Ohashi, MD, PhD, a professor at Jikei University in Japan, said in a press release.
Fabry disease is caused by mutations in the GLA gene, which lead to a defective alpha-galactosidase A enzyme. As a result, the enzyme cannot break down globotriaosylceramide (GL-3), a lipid, that then builds up in the lysosomes of patients’ cells.
Galafold restores enzymatic activity and clears GL-3 buildup in people who have GLA mutations amenable to the therapy, which accounts for 35 to 50 percent of all Fabry patients worldwide.
Japanese approval follows that in Europe and other countries, and was based on positive results from the randomized Phase 3 FACETS clinical trial (NCT00925301) in patients who had not received enzyme replacement therapy, a follow-up open-label Phase 3 study (NCT01458119), and the Phase 3 ATTRACT study (NCT01218659) in patients who had previously received enzyme replacement therapy.
“The Japanese approval for Galafold is a major step forward for more than 800 people currently known to be living with Fabry disease in Japan. We believe a significant portion of these Fabry patients have amenable mutations that are suitable for treatment with this differentiated precision oral therapy,” said John F. Crowley, chairman and CEO of Amicus Therapeutics Inc.
Hisao Harada, chairman of the Japan Fabry Disease Patients and Family Association, also noted that the approval of Galafold provides the first new treatment option for the disease in Japan in more than a decade.
In the U.S., Galafold recently received a priority review by the U.S. Food and Drug Administration, with a decision on approval expected before August 2018.