CAMBRIDGE, Mass. (AP) — Ariad Pharmaceuticals Inc. said Monday that U.S. and European regulators have given “orphan drug” status for a potential leukemia treatment it is developing.
Orphan drug status is given to drugs aimed at rare conditions or conditions that have a lack of treatments on the market. Incentives in the U.S. include seven years of market exclusivity following FDA approval, assistance in clinical trial design, a reduction in user fees, and tax credits.
The U.S. Food and Drug Administration gave the designation for the treatment of chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia. The European Medicines Agency gave it orphan drug status for chronic myeloid leukemia and acute lymphoblastic leukemia.
In the U.S., a product can be designated an orphan drug if it is meant to treat an illness than affects fewer than 200,000 people. In Europe, it goes to drugs for diseases affecting five out of every 10,000 people.
Ariad said it expects to move the compound, labeled AP24534, from early-stage testing into a registration trial later this year.
