Cullinan Therapeutics, Inc. (NASDAQ: CGEM) has received Orphan Drug Designation from the U.S. Food and Drug Administration for its investigational drug CLN-049, aimed at treating relapsed/refractory acute myeloid leukemia (AML).
CLN-049 is a novel FLT3xCD3 T cell engager currently undergoing a Phase 1 clinical trial to assess its safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy in patients with relapsed/refractory AML.
The FDA’s Orphan Drug Designation is granted to drugs and biologics intended for rare diseases affecting fewer than 200,000 people in the U.S.
Jeffrey Jones, MD, MBA, Chief Medical Officer, Cullinan Therapeutics, commented, “FDA Orphan Drug Designation for CLN-049 emphasizes both the urgent need for new therapies for people living with relapsed or refractory acute myeloid leukemia – including patients with TP53-mutated AML who currently face a particularly poor prognosis – and the potential of this FLT3-directed T cell engager to expand treatment options across the broadest population of AML patients.”
 received FDA Orphan Drug Designation for CLN-049, a drug targeting relapsed/refractory AML.){kind=link}