Rare Daily Staff

The U.S. Food and Drug Administration has granted BridgeBio subsidiary QED Therapeutics Fast Track designation and for its experimental drug infigratinib for the treatment of cholangiocarcinoma in adults with first-line advanced or metastatic cholangiocarcinoma.

The agency also granted the company Orphan Drug designation for infigratinib for the treatment of cholangiocarcinoma.

Cholangiocarcinoma is a rare and often fatal cancer of the bile ducts of the liver, which affects approximately 20,000 people in the United States and European Union each year. Treatment options are limited, and the five-year survival rate is only 9 percent.

Infigratinib is an orally administered, FGFR1-3 selective tyrosine kinase inhibitor that has shown activity in clinical studies, such as overall response rate in patients with chemotherapy-refractory cholangiocarcinoma with FGFR2 (fibroblast growth factor receptor 2) fusions and advanced urothelial carcinoma with FGFR3 genomic alterations. FGFR2 genetic aberrations are present in approximately 15 percent to 20 percent of people who have cholangiocarcinoma.

QED reported that enrollment is ongoing and patient dosing has started in the PROOF trial, a phase 3 clinical trial evaluating oral infigratinib in adults for first-line treatment of advanced cholangiocarcinoma with FGFR2 gene fusions or translocations.

The FDA’s Fast Track program is designed to facilitate the development and expedite the review of drugs with the potential to treat serious conditions and address an unmet medical need. Companies that receive Fast Track designation are provided the opportunity for more frequent interactions with FDA during clinical development and are eligible for accelerated approval and/or priority review, if relevant criteria are met.

The FDA grants Orphan drug designation to drugs or biologics intended to treat rare diseases or conditions. The designation allows the drug developer to be eligible for a seven-year period of U.S. marketing exclusivity upon approval of the drug, if the drug receives the first FDA approval for the rare disease or condition, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

“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,” said Susan Moran, chief medical officer for QED. “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 PROOF trial will enroll approximately 384 patients with first-line cholangiocarcinoma with FGFR2 fusions or translocations, as determined by molecular profiling. The primary endpoint is progression-free survival compared to standard of care chemotherapy (gemcitabine and cisplatin). Patients will be randomized 2:1 to infigratinib versus standard of care.

“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,” said Stacie Lindsey, president of the Cholangiocarcinoma Foundation. “Having a crossover option is very significant to patients and including it in the design of this trial demonstrates that QED is listening to them.”

Infigratinib received Fast Track designation for first-line treatment of adult patients with unresectable locally advanced or metastatic cholangiocarcinoma with FGFR2 gene fusions or translocations.

Photo: Susan Moran, chief medical officer for QED

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