If you’re not in the biotechnology industry, you may not be familiar with Amicus Therapeutics; they’re recognized as global leaders when it comes to developing therapies for orphan diseases. Amicus Therapeutics has a proprietary platform of medicines and technologies to treat those who are living with rare, and in many cases, life altering diseases. Some of these diseases include Lysosomal Storage Disorders, which encompasses Fabry disease and Pompe disease; in addition, Amicus Therapeutics provides treatment for genetic connective skin disorders like Epidermolysis Bullosa. Amicus Therapeutics relies on inventive science and clinical programs to treat their patients; in 2016, Amicus announced that Migalastat, a drug intended to treat Fabry disease, was approved by the European Union after undergoing two phase III clinical trials, and would be incorporated into their drug therapies.
HOW AMICUS THERAPEUTICS MAKES A DIFFERENCE
Amicus Therapeutics is based in Cranberry, NJ, and is committed to making a meaningful difference in the lives of their patients, as well as caregivers (https://www.dialdish.com/amicus-therapeutics-fabry-disease/). Founded in 2002, the company places a strong emphasis on developing enzyme replacement therapies and has worked in collaboration with top-tier pharmaceutical companies, like GlaxoSmithKline and JCR Pharmaceuticals in formulating an effective drug therapy to treat deficiency in the enzyme known as alpha-galactosidase. In fact, in 2008 Amicus opened a second facility in San Diego, as a means to further their research and broaden their operation.
HOW GRANTS HELP TREAT ORPHAN DISEASES
The work done by Amicus Therapeutics has caught the attention of not only their contemporaries but also Hollywood celebrities; in 2010, they received a grant totaling $500,000 from the Michael J. Fox Foundation (AliveNewsPaper). The funds were intended to support collaborative studies with the David Geffen School of Medicine. In addition, they received a grant for over $200,000 from the Alzheimer’s Drug Discovery Foundation, which was intended to support clinical work.
Why do orphan diseases receive grants? To better understand the reasoning behind this, we need to address what an orphan disease is; an orphan disease is a disease that affects less than 200,000 people. Since only a small percentage of people are affected by these diseases, there is no financial incentive for private companies to develop and market drugs to treat them, and therefore companies like Amicus Therapeutics has to rely upon grants.