Amicus Therapeutics announced the beginning of the availability of Galafold (migalastat) to treat Fabry disease in Japan, making this the first oral precision medicine available for patients with an amenable mutation in that country.
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 type of fat, that then builds up in the lysosomes of patients’ cells.
Galafold restores enzymatic activity and clears GL-3 buildup, and is suitable for patients 16 years or older who have a confirmed diagnosis of Fabry disease and a GLA mutation amenable to the therapy. Currently, amenable mutations account for 35 to 50 percent of all Fabry patients worldwide.
Healthcare providers in Japan can now access the Galafold Amenability Assay table to quickly identify which mutations are amenable to the treatment. To date, the assay has been used to classify more than 1,000 known GLA mutations as “amenable” or “not amenable” to treatment with Galafold. Updates will be submitted to the Galafold assay website as additional GLA mutations are identified and tested, the company announced in a press release.
“I welcome the introduction of Galafold as an oral precision medicine and the first new Fabry treatment option in more than a decade for a number of Fabry patients in Japan who have amenable mutations,” said Hisao Harada, chair of the Japan Fabry Disease Patients and Family Association (JFA).
Galafold was approved by Japanese regulatory authorities, at 123 mg capsules, under the priority review scheme for orphan drugs in March.
The approval was based on data from completed clinical trials of Galafold, including two Phase 3 studies (FACETS, NCT01218659, and ATTRACT, NCT01218659) in both treatment-naïve and enzyme replacement therapy switch patients, as well as a Phase 1 study that evaluated the pharmacokinetics of Galafold in Japanese volunteers.
“The launch of Galafold presents a differentiated treatment option that is good news for many Fabry patients in Japan. Significant unmet need remains for people living with Fabry disease, and I look forward to offering a new choice for patients with amenable mutations. Galafold has a unique mechanism of action that has demonstrated compelling results in naïve and treatment-experienced Fabry patients who have amenable mutations,” said Toya Ohashi, professor at Jikei University.
The European Commission approved Galafold as a first-line therapy for the long-term treatment of Fabry disease patients 16 years and older with an amenable mutation, opening it to use throughout the European Union.
The medicine is not yet approved in the United States, but the U.S. Food and Drug Administration (FDA) agreed to give priority review to Galafold in February, with a decision expected by August.
Outside the European Union and Japan, Galafold is also approved for use in Australia, Canada, Israel, South Korea and Switzerland.