FDA Grants Orphan Drug Designation to Cullinan’s CLN-049 in Relapsed/Refractory AML
The FDA granted Orphan Drug Designation to Cullinan Therapeutics’ CLN-049 for relapsed/refractory AML. CLN-049 is in Phase 1 studies and had already received Fast Track designation.
The U.S. Food and Drug Administration has granted Orphan Drug Designation to CLN-049, a novel, investigational FLT3xCD3 T cell engager, for the treatment of relapsed/refractory acute myeloid leukemia. The designation follows Fast Track designation for the treatment of relapsed/refractory AML and comes as the company continues an ongoing Phase 1 program.
CLN-049 is designed to target FLT3-expressing leukemia cells, offering a new immunotherapeutic approach for treating acute myeloid leukemia and myelodysplastic syndrome. CLN-049 binds to both mutated and non-mutated FLT3, enabling targeted action regardless of FLT3 mutational status, making the investigational treatment widely applicable to a broad population.
CLN-049 is being studied in a Phase 1, open-label, multicenter, first-in-human, multiple ascending dose study evaluating safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of intravenously administered CLN-049 in patients with relapsed/refractory AML or MDS (NCT05143996) and in a parallel Phase 1, open-label, dose escalation and dose expansion study for the treatment of patients with AML with measurable residual disease (EUCT 2023-506572-27-00).
The FDA’s Orphan Drug Designation provides orphan status to drugs and biologics intended to prevent, diagnose, or treat rare diseases or conditions that affect fewer than 200,000 people in the United States. Orphan Drug Designation qualifies sponsors for certain development incentives, including tax credits for qualified clinical trials, exemption from certain FDA user fees, and the potential for seven years of market exclusivity in the United States following marketing approval.
Acute myeloid leukemia is the most common form of acute leukemia in adults. Each year in the United States, approximately 22,000 people are diagnosed with AML, and about half as many lives are lost to the disease; globally, AML affects an estimated 144,000 people annually, with approximately 130,000 deaths. Outcomes for patients with relapsed or refractory disease remain poor, where five-year survival is 10% or less, and there are currently no approved immunotherapies for AML.