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FDA grants orphan drug designation to H3B-8800 for leukemia
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The FDA granted orphan drug designation to H3B-8800 for the treatment of patients with acute myeloid leukemia and chronic myelomonocytic leukemia, according to the drug’s manufacturer.
Phase 1 clinical trials of H3B-8800 (H3 Biomedicine, Eisai) — a potent, selective and orally bioavailable small molecule modulator of wild-type and mutant splicing factor 3b complexes — are ongoing.
Preclinical studies showed H3B-8800 induced dose-dependent modulation of canonical and aberrant splicing when given orally at tolerated doses.
In addition, oral administration of the agent demonstrated preferential antitumor activity in several preclinical xenograft models with spliceosome mutations.
“Receiving the orphan drug designation for H3B-8800 is a critical milestone for H3’s ongoing cancer genomics driven drug discovery program,” Markus Warmuth, MD, president and CEO of H3 Biomedicine, said in a company-issued press release. “We are pleased with the progress our scientific and clinical teams are making, and look forward to continue investigating H3B-8800 as a potential treatment option for patients with these diseases.”
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.