ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) 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,” 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.
About AP24534
ARIAD’s second oncology product candidate, AP24534, is an investigational pan-BCR-ABL inhibitor that has broad potential applications in cancer. ARIAD is currently completing a Phase 1 clinical trial of oral AP24534 in patients with advanced hematological cancers and plans to begin a pivotal registration trial of AP24534 later this year. Clinical proof-of-concept data from the Phase 1 trial provide clinical evidence of hematologic, cytogenetic and molecular anti-cancer activity of AP24534 in heavily pretreated patients with resistant and refractory chronic myeloid leukemia (CML), including those with the T315I mutation. In preclinical studies, AP24534 has also demonstrated potent inhibition of kinase targets associated with acute myeloid leukemia, as well as proliferation and angiogenesis in multiple solid tumors.
About CML and Ph+ ALL
CML is characterized by an excessive and unregulated production of white blood cells by the bone marrow due to a genetic abnormality that produces the BCR-ABL protein. After a chronic phase of production of too many white blood cells, CML typically evolves to more aggressive phases such as accelerated or blast crisis. Ph+ ALL is a subtype of acute lymphoblastic leukemia that also carries the Ph+ chromosome that produces BCR-ABL. It has a more aggressive course than CML and is often treated with a combination of chemotherapy and tyrosine kinase inhibitors. Because both of these diseases express the BCR-ABL protein, this would render them potentially susceptible to treatment with AP24534.
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ARIAD