The U.S. Food and Drug Administration granted accelerated approval to Cranbury-based Amicus Therapeutics for Galafold, an oral treatment for adults with Fabry disease.
Fabry disease is a rare genetic disorder caused by a defective gene that causes an enzyme deficiency that in turn causes a buildup of abnormal deposits of a fatty substance in the body. Symptoms include body ache, fatigue and burning pain of limbs; and complications include kidney failure and heart blockages.
Galafold is the first new treatment for Fabry disease in over 15 years and first oral treatment ever approved.
“The Fabry disease community has had an active voice in every stage of development of this medicine. We are grateful to this wonderful and passionate community, particularly the patients and physicians who have made this research possible through their participation in the clinical trials, as well as to the U.S. regulators and our ever-persistent and dedicated Amicus team,” said Amicus CEO John Crowley in a statement.
Galafold’s approval is conditional upon a requirement for Amicus to continue to study the medicine in a confirmatory Phase 4 program. A Phase 3 study found that the most common side effects of the medicine were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia.
Approximately 3,000 people in the U.S. are living with Fabry disease, half of whom currently are untreated.
With accelerated approval, Amicus will launch Galafold immediately. It will begin shipping to a limited distribution network next week.