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FDA grants orphan drug status to Actimab-A for AML in elderly patients
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The FDA granted orphan drug designation to a radiolabeled antibody for the treatment of acute myeloid leukemia in patients older than 60 years, its manufacturer announced recently.
Based on preclinical research, Actimab-A (Actinium) — lintuzumab linked to the actinium-225 payload — has demonstrated significant potential in its safety and efficacy profile, as well as potential potency, specificity and ease of use in an older population.
Older patients with AML are already at increased risk over younger patients, whereas patients with secondary AML are considered to have the more severe and less treatable form of AML, and thus shorter expected survival.
The FDA based its decision in part on preliminary data from an ongoing phase 1/2 multicenter clinical study, which found that median OS of seven patients with secondary AML — with prior myelodysplastic syndrome — was 9.1 months. This outcome compared favorably with historical standards of 2 to 5 months OS, depending on the treatment modality.
“The FDA’s decision to grant orphan drug status for Actimab-A is a significant milestone for the Company and recognizes the need for innovative new approaches to treat AML,” Kaushik J. Dave, PhD, president and CEO of Actinium, said in a press release. “The designation will provide Actinium access to various development benefits and financial incentives from the Agency, including an exemption from prescription drug user fees for Actimab-A for this indication and, if the drug receives marketing approval, it will enjoy 7 years of market exclusivity in the United States.”
Actinium noted that it expects additional updates to its phase 1/2 clinical trial in December.