Amicus Therapeutics uses the latest medical technology to come up with the best treatment for the rarest conditions. Based in Cranbury New Jersey; it’s an American public biopharmaceutical that the primary goal is to offer advanced therapies to the many orphans and rare, devastating diseases. The company under NASDAQ trading went public in 2007 under the trading symbol FOLD. Amicus Therapeutics has a robust development to treat human genetic diseases. Their primary interest of specialization is on lysosomal storage disorder and the development of Chaperone Advanced Replacement-Therapy which over the years has concentrated on enzymes replacement therapies. Amicus in 2014 was noted for having the broadest portfolio of pharmacological chaperones which are the smallest molecule (http://www.gcreport.com/amicus-therapeutics-announces-new-drug-application/).
The company leading product migalastat is in its final stages of development, and it’s a personalized medicine that treats those suffering from Fabry diseases. It uses a simple method to treat the disease using the genetic diagnosis. The medication also aims at stabilizing the endogenous mutant utilizing the alpha-galactosidase (Crunchbase). Amicus Therapeutics has done numerous collaborations with JCR Pharmaceuticals and GlaxoSmithKline to study alpha-galactosidase recombinant with coformulation, and it took the companies three years to do their study from 2010-2013.
Amicus Therapeutics has expanded its territories, and in 2008 they opened their office in San Diego which became their second research site. From 2013-2015 the company acquired two companies. First, it bought Callidus Biopharma its competitor and in the process added proprietary materials. Additionally, they were able to get an intellectual property that helped them continue with their enzyme replacement therapy to help treat Pombe disease. In September 2015 for a tune of $ 947 million in stock and cash they acquired Scioderm.
Amicus Therapeutics uses three fundamental characteristics to invest in their programs which including using the best clinical data to find meaningful treatment for the patients, to use the latest technology and find the best treatment for a sporadic condition.