European Regulatory Committee Recommends Orphan Medicinal Product Designation For Amicus Therapeutics, Inc.' Amigal(TM) For Fabry Disease

CRANBURY, N.J., April 11 /PRNewswire/ -- Amicus Therapeutics, a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of human genetic diseases, today announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMEA) has recommended orphan medicinal product designation for Amigal(TM) (migalastat hydrochloride or AT1001).

Amigal is being developed as an oral therapy for the treatment of Fabry disease, a lysosomal storage disorder caused by an enzyme deficiency that leads to pain, kidney failure, and an increased risk of heart attack and stroke. Amigal is currently being studied in Phase II clinical trials in the U.S., the U.K., Brazil, and Australia.

Fabry disease affects approximately 5,000 to 10,000 people in the developed world, but recent evidence suggests that the disease may be significantly underdiagnosed. Orphan designation granted by the EMEA provides 10 years of market exclusivity after approval and other incentives to support and encourage development of drugs for diseases that affect fewer than five in every 10,000 people in the European Union (EU). Fabry disease affects an estimated 1.75 out of every 100,000 people in the EU. In addition, for diseases with an approved therapy in Europe, such as Fabry disease, a new therapy must have the potential to provide significant benefit over the existing therapy in order to receive orphan designation. The recommendation now goes to the European Commission for approval.

In February 2004, the Food and Drug Administration's Office of Orphan Products Development granted orphan designation for Amigal in the United States.

About Amicus Therapeutics

Amicus Therapeutics is a biopharmaceutical company based in Cranbury, New Jersey, developing small molecule, orally-administered pharmacological chaperones for the treatment of human genetic diseases. Many of these diseases are the result of missense and other genetic errors that cause the misfolding and degradation or accumulation of a particular protein. Amicus' products act as pharmacological chaperones that selectively bind and "rescue" the misfolded target protein to restore its proper conformation and natural function, which in turn restores the function of the affected cells. Amicus' lead compound, Amigal(TM), is in Phase II clinical trials for Fabry disease. AT2101 for the treatment of Gaucher disease is expected to enter clinical studies in the first half of this year. The company is also developing programs for additional genetic diseases.

Additional information about Amicus can be found at http://www.amicustherapeutics.com.

CONTACTS: Corporate: Media: Matthew Patterson Stephen Gendel Amicus Therapeutics GendeLLindheim BioCom Partners (609) 662-2000 (212) 918-4650

CONTACT: Corporate: Matthew Patterson of Amicus Therapeutics,+1-609-662-2000; or Media: Stephen Gendel of GendeLLindheim BioComPartners, +1-212-918-4650, for Amicus Therapeutics

Web site: http://www.amicustherapeutics.com/