If you’re unfamiliar with Amicus Therapeutics, it is an American biopharmaceutical company, headquartered in New Jersey, and is predicated on fighting orphan diseases. What are orphan diseases? They are categorized as rare, and in some cases, life-altering illnesses that do not receive the same level of research or drug treatment options as other diseases. These diseases include, but are not limited to, Lysosomal Storage Disorders and skin disorders like Epidermolysis Bullosa. If you haven’t heard of these diseases, it’s because they’re not as prevalent others; however, they still warrant research, and those who are afflicted will need to be treated, which is why we need companies like Amicus Therapeutics.
Since Amicus Therapeutics is not privy to the same types of clinical trials, or receive the same level of support from pharmaceutical companies, the biopharmaceutical company must rely on innovative science and customized clinical programs, to treat those with orphan diseases. Through the effective use of science and these clinical programs, Amicus Therapeutics has received approval from the European Union to use Migalastat (Galafold) as a drug treatment option for Fabry disease (one of many orphan diseases). This approval was hard fought, as Migalastat did not receive approval until after it had undergone two rigorous phase three clinical trials.
Amicus Therapeutics was found in 2002 and is committed to working with pharmaceutical companies, and developing enzyme replacement drugs like Migalastat. This commitment has led to the opening of a second facility based in San Diego, CA, which was opened in 2008 (YahooFinance). The New Jersey and the San Diego locations work collectively with companies like JCR Pharmaceuticals and GlaxoSmithKline, in developing, even more, drug therapies aimed at treating enzyme deficiencies, that contribute to orphan diseases.
Thankfully, much of the work done by Amicus Therapeutics has caught the attention of some of Hollywood’s biggest names, allowing the company to continue their research and help hundreds of people. In fact, in 2010 Amicus Therapeutics received a generous grant from actor Michael J. Fox totaling $500,000. These grants are a tremendous blessing and will allow Amicus Therapeutics to continue doing the amazing work that they do.