How Amicus Therapeutics Is Addressing the Significant Unmet Needs for Rare and Orphan Diseases

Amicus Therapeutics has been at the forefront of individualized medicine in the biotechnology industry. The company focuses on rare and orphan diseases treatment using technologies that are science driven to deliver ultimate benefits to the patients. Amicus believes that positioning themselves at the forefront of developing therapies for unmet needs it the only way to help the vast majorities of patients with rare devastating diseases. Therefore, the company has a robust development pipeline of other treatments for different genetic diseases.



As a global biotechnology company, Amicus Therapeutics has unique platform technologies in development for people with disorders such as lysosomal storage disorders (LSDs), and Pompe disease as well as skin disorders such as the epidermolysis Bullusa. Precisely, Amicus product development is based on advanced replacement therapy platform and concentrates on ERTs (Enzyme replacement therapies). In the recent years, the company has been noted to have the broadest portfolio of pharmacological chaperones in the industry (YourBeautyCraze).



Amicus Therapeutics has recently advanced its precision product Migalastat, in treating patients with Fabry disease (alpha-galactosidase A deficiency) a rare disorder where sphingolipids are not well metabolized. Previous Fabry disease therapies involved using ERT to manage the ailment, but Amicus Therapeutics therapy does not replace the enzymes, rather it helps the ones that are produced to work better ( The company estimates that more than half of Fabry disease patient could benefit from this treatment.The company has also collaborated with GlaxoSmithKline as well as JCR pharmaceutical to investigation preclinical programs in other rare diseases such as the CDKL5 deficiency.



In 2009, Amicus faced a major financial setback after a termination of an agreement with The Shire, which led to a major corporate reconstruction. However, the company has consistently innovated new ERT products for Pompe diseases, LSD and Fabry diseases. Amicus therapeutics center of inventive science is the need of patients with rare diseases, so the company continues to develop new products that will make the difference in the lives of these patients as well as their caregiver.


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