Amicus Therapeutics is a biopharmaceutical company founded in 2002 based in Cranbury New Jersey and also has a research facility in San Diego. The company uses FOLD as its trading symbol has a global reach with funding partners such as the Alzheimer’s Drug Discovery Foundation and the Michael J. Fox foundation among others. The company focus is on the development of precision medications for devastating, orphan and rare ailments especially those brought about by a genetic mutation. Using CHART-Chaperone advanced replacement Therapy for product development, Amicus Therapeutics has developed enzyme replacement therapies-ERTs products for disorders termed as lysosomal storage disorders.
Among the rare diseases that Amicus Therapeutics has developed medicines and technologies for include; Pompe disease, Fabry disease, and Epidermolysis Bullosa. The company goal is to bring a meaningful difference to the lives of the patients with rare and devastating diseases and also to their caregivers. Amicus Therapeutics primary objective is to meet the rare diseases community needs. It has been on the forefront in providing medicines for rare ailments and in 2014 the company was recognized in the pharmaceutical industry as having the largest molecular pharmacological chaperones collection.
Migalastat The New Drug For Fabry Disease
Recently, Amicus Therapeutics announced that it has been given the go ahead by FDA to submit migalastat a new precision medication for Fabry’s disease under Subpart H. The submission will be done in the fourth quarter of this year. The medication is an oral drug that can only be administered to teenagers and adults aged 16 years and above. The new drug application will be based on collected data from clinical trials that show that the drug reduces GL-3. GL-3 causes increased morbidity of Fabry’s diseases over time leading to pain, stroke, heart diseases and kidney failure for the patients. Today the number of people suffering from Fabry’s diseases in the US alone is almost 3000 and Amicus Therapeutics believe that this number is big enough for the development of migalastat (WeeklyOpinion). Amicus Therapeutics hopes the drug will be approved soon so that it can change the lives of these patients.
In addition to migalastat their lead product, Amicus Therapeutics have developed other products including SD-101 which is a potential therapy product for Epidermolysis Bullosa a connective genetic disorder. This product is also in its late stages of development. They also have a biologics program ATB200/AT2221 that should be combined with a pharmacological chaperone in its administration. It is for patients suffering from Pompe Disease. In a world where false medication has risen especially for rare diseases. Amicus Therapeutics continues to set new levels of achievements in its specialty and changing the lives of those suffering from devastating and rare diseases.
More about Amicus Therapeutics at https://finance.yahoo.com/quote/FOLD?ltr=1