Amicus Therapeutics is a publicly listed biotechnology company focused on the discovery, development, and delivery of treatments for rare metabolic diseases. Headquartered in New Jersey, the company also operates officers internationally. They include the UK, Canada, Japan, Germany, France, Italy, The Netherlands, Spain, and Australia.
Amicus' developments include one commercialized medication for Fabry disease, an investigational enzyme replacement therapy (AT-GAA) in late stage development for the treatment of Pompe disease, and several gene therapy pipeline and growth platform for lysosomal storage disorders, including Batten Disease, Neimann-Pick Type C, Wolman Disease, and Tay Sachs.
In August 2018, the US FDA approved Amicus Therapeutics' Galafold, the first oral therapy to treat Fabry disease, a rare and sometime fatal condition in which accumulation of fat damages several organs. It is a long-term treatment for adult patients with specific genetic mutations, which account for an estimated 35 to 50% of diagnosed cases globally. Chemically known as migalastat, the drug has also been approved in Europe, Japan, Australia, and Canada.
The company's investigational therapy, AT-GAA, for Pompe disease, an inherited Lysosomal Storage (LSD) caused by a deficiency of the enzyme acid alpha-glucosidase (GAA), completed a Phase 2 safety and pharmacokinetics study in 2013.
Amicus is also conducting multiple Phase 1/2 studies for their CLN6 gene therapy to treat Batten disease, a broad class of rare, fatal, inherited disorders of the nervous system known as neuronal ceroid lipofuscinoses (NCLs).
On July 25, 2002 Amicus Therapeutics completed their series A funding round with $2 million in funding from CHL Medical Partners.
On May 13, 2004 Amicus Therapeutics completed their series B funding round with $31 million in funding from Canaan Partners (lead investor), Radius Venture Partners, New Enterprise Associates, Frazier Healthcare Partners, and CHL Medical Partners.
On September 8, 2005 Amicus Therapeutics completed their series C funding round with $55 million in funding from Quaker BioVentures (lead investor), Radius Ventures, Prospect Venture Partners, Palo Alto Investors, New Enterprise Associates, Garden State Life Sciences Venture, Frazier Healthcare Partners, Canaan Partners, and CHL Medical Partners.
Amicus Therapeutics receives U.S. approval for Fabry disease drug