Amicus' Amigal gets OD nod from COMP

New Jersey, USA-based biopharmaceutical company Amicus Therapeutics, a specialist developer of oral drugs for the treatment of human genetic diseases, says that the European Medicines Agency's Committee for Orphan Medicinal Products has recommended that its drug Amigal (migalastat hydrochloride) be granted Orphan Drug designation for the treatment of Fabry's disease.

The condition is a lysosomal storage disorder which causes kidney failure and increases the risk of heart attack and stroke. Fabryl interacts with the malformed enzymes which cause the condition, allowing them to regain their correct function.

Currently, Genzyme's Fabrazyme (agalsidase beta) is the leading treatment for the condition, having been approved in the European Union in 2001. As a result, Amicus' drug, which is passing through Phase II clinical trials, must demonstrate significant benefit over Genzyme's enzymatic therapy in order to receive OD status.