FDA Grants Orphan Drug Designation to ASLAN003 in Acute Myeloid Leukemia
The U.S. Food and Drug Administration (FDA) has granted ASLAN003 Orphan Drug designation as a treatment for acute myeloid leukemia (AML). ASLAN003 is an orally active, potent inhibitor of human dihydroorotate dehydrogenase (DHODH) that has the potential to be a first-in-class drug in AML.
The FDA grants Orphan Drug designation to agents intended to treat a rare disease or condition that affects fewer than 200,000 individuals in the United States. Patients with relapsed or refractory AML represent the majority of the total AML population. In 2016, the annual incidence of those with relapsed/refractory AML was approximately 13,000 patients in the United States.
In previous clinical studies, ASLAN003 has demonstrated potent inhibition of DHODH (up to two orders of magnitude stronger than first-generation DHODH inhibitors), lack of toxicities associated with first-generation inhibitors and other novel AML therapies, and the potential to induce differentiation in blast cells and applicability in a broad range of AML patients. A phase II clinical trial of ASLAN003 in AML is being conducted in Asia, and researchers expect to report interim data in the second half of 2018.
The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.
