Amicus Therapeutics, a global biotechnology company was founded in 2002 in Cranbury, NJ. Its primary focus is to develop therapies for genetic, rare and orphan diseases. They are unique in that as of 2014, Amicus Therapeutics had the widest grouping of small molecule pharmacological chaperones among their competitors in the industry. Amicus has developed therapies for some rare diseases, such as Lysosomal Storage Disorders Fabry disease (affecting 1 in 117,000 people) and Pompe disease (affecting 5,000 to 10,000 people worldwide) and the genetic connective skin disorder Epidermolysis Bullosa (affecting 20 in 1,000,000 infants). In fact, Amicus Therapies has established a productive pipeline that is continuously generating a steady flow of new and effective therapies to treat genetic disorders. Visit Yahoo Finance to learn more about Amicus Therapeutics.
Their most promising product is Migalastat, a medicine that treats Fabry disease. It is currently in late-stage development. Another product in late-stage development that would also be a first-to-market therapy is SD-101, a product that treats Epidermolysis Bullosa (EB). Amicus is using all their advanced techniques to create an enzyme replacement therapy for Fabry and Pompe disease. In addition, they hope to use similar therapies to treat other Lysosomal Storage Disorders (LSDs). Their program ATB200/AT2221 is a uniquely biologically engineered therapy to treat Pompe disease. It is to be used with a pharmacological chaperone.
The clinical trials that Amicus Therapeutics is currently conducting include one for treating the genetic mutations in the CDKL5 gene causing the CDKL5 protein deficiency. The CDKL5 protein deficiency causes a disorder that manifests clinically as persistent seizures and severe impairment in neurological development, all occurring in infancy. As a consequence these children cannot walk, or perform basic self maintenance and they may also have scoliosis, visual impairments, sensory issues, and gastrointestinal complications.
Amicus Therapeutics acquired Callidus Biopharma, a competitor in 2013 and Scioderm, Incorporated, a rare disease company in 2015. Both acquisitions have put Amicus in a better position to release their first therapy. Amicus Therapeutics has its finger on the release of some very important therapies for several rare diseases. Their successful development and release will finally make the lives hundreds of children and adults more livable. View the market summary of Amicus Therapeutics at finance.google.com.