Carna Biosciences Receives FDA Orphan Drug Designation for Monzosertib in AML

TOKYO, Jul 21 (Pulse News Wire) – Carna Biosciences,inc. (4572.T) announced that it has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for monzosertib (AS141) as a treatment for acute myeloid leukemia (AML).

AML is a rare blood cancer characterized by abnormal proliferation of white blood cells in the bone marrow, with significant unmet medical needs among patients who relapse or fail existing treatments. Monzosertib, developed by Carna Biosciences, is a highly selective CDC7 inhibitor being studied as a novel therapeutic approach independent of specific genetic mutations. Currently, the company is collaborating with MD Anderson Cancer Center to advance clinical development of a three-drug combination therapy involving azacitidine (AZA) and venetoclax (VEN) for relapsed or refractory AML patients.

"The receipt of ODD marks a significant milestone in our efforts to address the high unmet medical need in AML," said President and CEO Yoshiro Yoshino. "We aim to continue advancing the clinical development of monzosertib to contribute to resolving the unmet medical needs of AML patients." This designation provides benefits such as enhanced consultation opportunities with the FDA, reduced approval fees, and post-market exclusivity. It does not impact the company's fiscal 2026 consolidated earnings forecast.