BridgeBio Pharma Inc.'s infigratinib for treating a rare type of cancer received fast-track and orphan-drug designations from the U.S. Food and Drug Administration.
The drug, developed by the Palo Alto, Calif.-based biopharmaceutical company's unit QED Therapeutics Inc., received an orphan-drug tag for its treatment of cholangiocarcinoma, a rare type of cancer that develops in the bile ducts, which connect the liver to the gallbladder and small intestine. These ducts carry the digestive fluid called bile.
Additionally, infigratinib received a fast-track designation as an initial treatment for adult patients of a form of cholangiocarcinoma that spreads across tissue and cannot be treated surgically.
The drug targets susceptible genetic alterations in patients' fibroblast growth factor receptors, which regulate important biological processes including cell growth and division during development and tissue repair.
Cholangiocarcinoma affects about 20,000 people in the U.S. and EU each year, with only 9% of patients surviving for five years with the cancer.
Orphan-drug designation encourages companies to develop cures for rare diseases by offering them tax reductions, fee exemptions and a license to be the sole sellers of their treatment for seven years.
The FDA's fast-track program is designed to facilitate the development and review of new drugs intended to treat life-threatening conditions. It allows for more frequent communications between the FDA and the company, which may also be eligible for priority review if certain criteria are met.
Infigratinib's effectiveness as initial treatment for the cancer is being tested in the late-stage Proof trial on 384 adult patients.