FDA grants orphan drug designation to SEL24/MEN1703 for AML
The FDA granted orphan drug designation to SEL24/MEN1703 for the treatment of acute myeloid leukemia.
SEL24/MEN1703 (Menarini Group) is a dual PIM/FLT3 inhibitor.
The phase 1/phase 2 DIAMOND-01 trial is evaluating the agent as monotherapy for patients with relapsed or refractory AML. Results of the dose-escalation portion of the trial showed SEL24/MEN1703 demonstrated a manageable safety profile up to the recommended dose of 125 mg daily, according to a Menarini Group-issued press release. Results of the cohort expansion portion of the study showed preliminary single-agent efficacy, especially for patients with IDH-mutant disease.
“FDA orphan drug designation represents an important milestone for SEL24/MEN1703 program” Elcin Barker Ergun, CEO of Menarini Group, said in the release. “SEL24/MEN1703 is a first-in-class, orally available, dual PIM/FLT3 inhibitor that can contribute to finding new treatment paradigms for AML, where significant unmet needs exist — especially as resistance develops in later lines. We look forward to advancing the clinical development of SEL24/MEN1703 in AML, with a goal to ultimately provide patients with a new therapeutic option for this hard-to-treat disease.”
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
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