FDA Grants Fast Track, Orphan Drug Designations to Infigratinib for Cholangiocarcinoma
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The FDA granted fast track designation to infigratinib for first-line treatment of patients with unresectable advanced or metastatic cholangiocarcinoma who harbor FGFR2 gene fusions or translocations, according to the agent’s manufacturer.
The agency also granted orphan drug designation to infigratinib (BridgeBio Pharma/QED Therapeutics) for the treatment of cholangiocarcinoma.
Cholangiocarcinoma — a cancer of the bile ducts — affects approximately 20,000 people in the United States and Europe each year. An estimated 15% to 20% of patients have FGFR2 genetic aberrations. Treatment options are limited, and 5-year survival is approximately 9%.
Fast track designation is intended to expedite the review of drugs with the potential to treat serious conditions and address unmet medical needs.
The FDA’s 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.
“We believe that fast track and orphan drug designations for infigratinib for the treatment of cholangiocarcinoma underscores the need for new, targeted treatments for genetically driven subsets of this cancer, particularly for adults with first-line advanced or metastatic cholangiocarcinoma,” Susan Moran, MD, MSCE, chief medical officer for QED, said in a company-issued press release. “Fast track designation will enhance our interaction with the FDA on our first-line advanced or metastatic cholangiocarcinoma program and may help us get this medicine to patients more quickly.”
The phase 3 PROOF trial is designed to evaluate oral infigratinib as first-line treatment of patients with advanced cholangiocarcinoma who have FGFR2 gene fusions or translocations.
Enrollment and patient dosing are underway. Investigators intend to randomly assign 384 patients 2:1 to infigratinib or standard chemotherapy with gemcitabine and cisplatin. PFS will serve as the primary endpoint.
“Importantly, in this trial, patients who are assigned to receive standard of care will be allowed to cross over and receive infigratinib if they do not respond to chemotherapy,” Stacie Lindsey, president of Cholangiocarcinoma Foundation, said in the press release. “Having a crossover option is very significant to patients.”