ARIAD Pharmaceuticals' investigational pan-BCR-ABL inhibitor receives FDA and EMA orphan drug designation

ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) today announced that its investigational pan-BCR-ABL inhibitor, AP24534, has been granted orphan drug designation by both the U. S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). In the U.S., the orphan designation of AP24534 is for the treatment of chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) and in the E.U., its orphan designation is for CML and acute lymphoblastic leukemia.

“While impressive medical gains have been made over the past decade in treating various forms of leukemia, it is clear that the disease can be severe and life-threatening when it progresses”

“While impressive medical gains have been made over the past decade in treating various forms of leukemia, it is clear that the disease can be severe and life-threatening when it progresses,” said Frank Haluska, M.D., Ph.D., vice president, clinical affairs of ARIAD. “Orphan drug designation for AP24534 highlights the lack of therapeutic options available for patients with CML or Ph+ ALL, who are resistant or refractory to currently available therapies. We expect to advance AP24534 into a pivotal registration trial later this year and pursue subsequent regulatory submissions for marketing authorization in these hematological cancers.”

Orphan drug designation, which is intended to facilitate drug development, provides substantial potential benefits to the sponsor, including funding for certain clinical studies, study-design assistance, and several years of market exclusivity for the product upon regulatory approval.