Amicus Therapeutics, Inc. (NASDAQ:FOLD) has revealed on Wednesday irs plans to submit a Japanese New Drug Application (J-NDA) for the commercialization of an oral medicine for Fabry disease, migalastat.
J-NDA Filing
Accordingly, the J-NDA will largely depend on the migalastat clinical trial results, which include a Phase 1 clinical trial evaluating the pharmacokinetics (PK) among Japanese subjects and two Phase 3 clinical trials. The Japanese Pharmaceuticals and Medical Devices Agency (PMDA) confirmed that these studies have met the J-NDA filing requirements. Consequently, Amicus Therapeutics will no longer be compelled to conduct further clinical trials in Japan.
John Crowley, Amicus Therapeutics Chairman and CEO, noted that the company is delighted with the faster-than-expected pace of the submission processing. He reiterated that he J-NDA filing is an integral part of the company’s global expansion strategies for migalastat. Crowley believes that many Fabry patients in Japan can benefit from the marketing approval of migalastat.
Migalastat for Fabry Disease
Fabry disease, a rare genetic condition, can potentially threaten a patient’s life due to the accumulation of globotriaosylceramide (GL-3) in the lysosome caused by a dysfunctional enzyme. With migalastat, such dysfunctional enzyme among those with amenable mutations is stabilized to clear the GL-3 accumulation.
Japan is presently the second biggest Fabry market globally, accounting for about 13% of the overall sales in 2015.
Migalastat Global Availability
Recently, Amicus Therapeutics has received a commercialization approval for migalastat from the European Commission (EC) based on two Phase 3 clinical trials and ongoing long-term extension clinical programs. Marketed as Galafold in Europe, the drug is prescribed to individuals aged 16 years old and above who are suffering from Fabry disease with amenable mutations. It is important to note that Galafold is not a potential treatment for Fabry disease patients who: 1) have non-amenable mutations; 2) have severe renal impairment; and 3) are 15 years old and below. It is also not highly recommended for women who are pregnant and planning or trying to get pregnant.
Meanwhile, approval for migalastat in the US is currently underway as the US Food and Drug Administration (FDA) conducts its review. An update relating to this effort will likely be issued by the third quarter.
