FDA grants orphan drug status to DKN-01 for certain gastrointestinal cancers
The FDA granted orphan drug designation to DKN-01 for the treatment of gastric or gastroesophageal junction cancers.
DKN-01 (Leap Therapeutics) is a humanized monoclonal antibody that blocks the activity of the Dickkopf-1 (DKK1) protein, a modulator of Wnt/Beta-catenin signaling.
“Orphan drug designation for DKN-01 in gastric and gastroesophageal junction cancer is another significant milestone in our DKN-01 development program and underscores the need for new treatment options for these indications,” Douglas E. Onsi, president and CEO of Leap Therapeutics, said in a company-issued press release. “We believe DKN-01 has the potential to be an important new therapy for this patient population that remains an area of high unmet medical need.”
Multiple trials are underway to evaluate DKN-01 for treatment of gastroesophageal, gynecologic, hepatobiliary and prostate cancers.
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.