July 2019, AxxessBio was granted an exclusive distribution right by Amicus Therapeutics to distribute Galafold® (migalastat) in Singapore.
Galafold® is the first and only oral therapy for adults with confirmed Fabry disease with an amenable GLA variant.
Fabry disease is an inherited disorder that causes build- up of fats in body cells. This is due to a mutation of a gene called GLA gene. This gene instructs the formation of an enzyme called alpha Gal A which breaks down the fats called globotriaosylceramide. Mutations in the GLA gene alter the structure and function of the enzyme, preventing it from breaking down fats effectively.
As a result, these build up in cells throughout the body, particularly cells lining blood vessels in the skin and cells in the kidneys, heart, and nervous system. The progressive accumulation of this substance damages cells, leading to the varied signs and symptoms of Fabry disease.
Amicus Therapeutics is a global, patient-dedicated biotech focused on discovering, developing, and delivering high-quality medicines for people living with rare metabolic diseases. For more information on the company, its products and pipeline, please visit https://www.amicusrx.com.