Amicus Therapeutics is an American therapeutics company based in Cranbury, New Jersey.
They’re new groundbreaking treatment announcement, however, has very little to do with America, or even New Jersey for that matter. The biopharmaceutical company has just announced that Health Canada has approved the treatment Galafold for adult patients with fabry disease.
Galafold is an oral precision medicine that will hopefully successfully serve as a long-term treatment for adult fabry patients with an amenable alpha-Gal A mutation. This treatment should be available to Canadian patients in the next few weeks.
This is pretty significant news, because like with any other disease, fabry patients are eager to receive new treatment options. Fabry disease results from the buildup of globotriaosylceramide (a particular type of fat) in the body’s cells. Of course, this buildup is not natural and leads to symptoms that affect many parts of the body which is characteristic of the inherited disorder.
Other features of fabry disease include pain in the hands and feet, a decreased ability to sweat, cloudiness in the eye, gastrointestinal system problems, and hearing problems like ringing in the ear and hearing loss. Along with fabry disease are some potentially life-threatening risks like progressive kidney damage, heart attack and stroke. To learn more about the frequency, genetic changes, inheritance pattern, diagnosis and management of fabry disease, click here.
Since the side effects and complications associated with fabry disease are so serious, Amicus Therapeutics expects this announcement to be great news for the Canadian fabry community.
“The approval of Galafold represents a significant step forward for the Canadian Fabry community. . . We look forward to launching Galafold in Canada to further advance our mission of expanding global access to Galafold for people living with Fabry disease who have amenable mutations,” said Amicus Therapeutics Chief Executive Officer and Chairman John F. Crowley.
Galafold was approved by Health Canada after releasing data from two crucial Phase 3 studies. The treatment works by stabilizing and correcting an enzyme in the body in a way that clears the buildup of disease in patients with amenable mutations. This treatment is not only projected to be highly successful, but also versatile within the fabry community.
“Galafold has a unique mechanism of action that is rooted in the underlying genetics of Fabry disease. With approximately 270 mutations identified as amenable to this chaperone therapy in Canada, there are many patients who could potentially benefit from this new treatment option,” said Dr. Daniel Bichet from the University of Montreal.
Needless to say, this announcement won’t only be a huge deal because it’s the first time in over a decade that a fabry disease treatment has been approved in Canada, but also because it has the potential to treat hundreds of different mutations too.
