- Amicus Therapeutics' Galafold (migalastat) is intended to treat Fabry disease, a rare genetic enzyme-deficiency disease in which patients experience lipid build-up that is painful and can result in kidney failure, heart disease or stroke.
- When Amicus announced that it would delay submission of Galafold, the stock fell by 53% and lost $760 million in market value. In early trading Monday, shares went back up by about 11%.
- Amicus intitally intended to submit to U.S. and E.U. authories by the second half of 2015.
Fabry disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A). According to estimates provided by Amicus, this disease affects roughly 5,000 to 10,000 people worldwide. Of that population, about 40% to 50% are not being treated. According to Amicus, approximately 30% to 50% of all patients with Fabry disease could benefit from Galafold, however, the company has to delay filing because of unexpected gastrointestinal (GI) symptoms.
Upon learning of these symptoms, the FDA requested that Amicus combine two clinical trials and recalibrate the data to reflect a modified study design. At the same time, Galafold is still on track in the E.U. to recieve approval by the end of this year, or early 2016.