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FDA grants orphan drug designation to OBI-3424 for acute lymphoblastic leukemia
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The FDA granted orphan drug designation to OBI-3424 for treatment of acute lymphoblastic leukemia, according to the agent’s manufacturer.
OBI-3424 (OBI Pharma Inc.) is a first-in-class DNA alkylating agent that targets solid tumors and blood cancers that overexpress aldo-keto reductase 1C3 (AKR1C3).
AKR1C3 overexpression has been observed in several difficult-to-treat malignancies, including ALL, hepatocellular carcinoma and castration-resistant prostate cancer.
Enrollment is underway at The University of Texas MD Anderson Cancer Center for a phase 1/phase 2 study of OBI-3424 for patients with HCC, castration-resistant prostate cancer and other solid tumors.
The FDA in July granted orphan drug designation to the agent for treatment of HCC.
“This additional orphan drug designation for OBI-3424 by the FDA is a significant step in the development of this drug candidate in ALL, including T-ALL, an unmet medical need disease with limited treatment options,” Amy Huang, general manager of OBI Pharma, said in a company-issued press release. “We are excited that the FDA has recognized the need to develop novel targeted therapeutic agents such as OBI-3424 in the fight against ALL.”
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.