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FDA grants orphan drug designation to ABTL0812 for pancreatic cancer
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The FDA granted orphan drug designation to ABTL0812 for the treatment of pancreatic cancer, according to the drug’s manufacturer.
“Receiving orphan drug designation for ABTL0812 (Ability Pharmaceuticals) is an important regulatory milestone achieved and an exciting step toward clinical advancement of this first-in-class therapy,” Carles Domenech, PhD, CEO of Ability Pharmaceuticals, said in a press release.
ABTL0812 is a first-in-class fully differentiated oral targeted anticancer compound that inhibits the PI3K/Akt/mTOR pathway.
In preclinical studies, ABTL0812 showed efficacy as a single agent and in combination with taxanes, platinum compounds and gemcitabine. Treatment induced tumor regression without increasing the toxicity associated with chemotherapy.
A phase 1/1b clinical trial — designed to evaluate ABTL0812 in 29 patients with pancreatic cancer — demonstrated that the drug was safe and well tolerated compared with other inhibitors of the pathway, and no dose-limiting toxicities were reported.
In addition, two patients showed 14-month and 18-month disease stabilization.
ABTL0812 is currently under phase 2 investigation as a first-line therapy in combination with chemotherapy in patients with endometrial or squamous cell lung cancer.
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.