Today, cases of human genetic illnesses are on the rise. Many individuals are suffering from serious rare and orphan disorders. Amicus Therapeutics, a biotechnological company based in Cranbury, New Jersey is very instrumental in fighting the genetic diseases. The brand is an American public biopharmaceutical corporation and has been on the forefront in the treatment of alarming rare and orphan conditions such as Pompe Disease, Fabry Disease, and Lysosomal Storage Disease (LSD).
Amicus Therapeutics s products are based on CHART (Chaperon Advanced Replacement Therapy) and ERTs (Enzyme Replacement Therapies). The company was founded in 2002 and is currently led by John Crowley, the CEO, Bradley Campbell, the COO, and William Baird, the CFO. In 2008, Amicus expanded from New Jersey and a new research site was opened in San Diego. This was to facilitate more production and market growth of the brand. In 2010, the organization received money grant worth $210,300 from Alzheimer Drug Foundation to help in the preclinical research in respect to Alzheimer treatment.
In 2013, Amicus Therapeutics procured Callidus Biopharma, a competitor. This gave them the proprietary and intellectual property right for ERT treatment practice against Pompe Disease. In 2015, Amicus also acquired Sciodern for $947 million. Generally, the company has experienced vital strides in its market operations. Their focus to fight devastating genetic illnesses makes them effective in the therapeutic work. They have set the bar high and many people look up to them for effective solutions to rare and orphan conditions.
The clinical program products from Amicus Therapeutics include:
- Amicus lead product known as Migalastat is effective in the treatment of Fabry Disease based on genetic diagnosis.
- SD-101 is effective in the treatment of Epidermolysis Bullosa (EB), a rare genetic tissue disease.
- ERT programs for treating Pompe Disease and other LSDs
In addition to the clinical programs, the company is currently doing research on CDKL5 (cycling-dependent kinase-like 5), a gene that affects brain development.
Generally, the clinical treatment products for Fabry Disease, EB, and Pompe Disease are a clear manifestation of the market effectiveness of Amicus Therapeutics in fighting rare and orphan disorders. The company is very instrumental in managing genetic illnesses globally.