FDA has granted accelerated approval to Pemazyre (pemigatinib), the first treatment approved for adults with certain types of previously treated, advanced cholangiocarcinoma.
FDA also approved the FoundationOne CDX (Foundation Medicine Inc.) as a companion diagnostic for patient selection.
Incyte Corp. sponsors the drug.
“This approval demonstrates that while we continue to focus our efforts on addressing the COVID-19 pandemic, the FDA remains committed to the important work of reviewing treatments for patients with cancer and other serious conditions,” said Richard Pazdur, director of the FDA Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, said in a statement.
“With Pemazyre, we considered the observed efficacy results to be clinically meaningful and the overall risk to benefit assessment for patients with tumors harboring FGFR2 gene fusions and other rearrangements to be favorable, particularly when we considered that these patients have no other good options following first line treatment with chemotherapy,” Pazdur said.
The approval is for locally advanced or metastatic cholangiocarcinoma in patients who have tumors that have a fusion or other rearrangement of the FGFR2 gene.
At diagnosis, a majority of patients with cholangiocarcinoma have advanced disease. Prior to the April 17 approval, there were no FDA-approved therapies for the disease. FGFR2 fusions have been found in the tumors of approximately 9% to 14% of patients with cholangiocarcinoma. Pemazyre is a tablet that works by blocking FGFR2 in tumor cells.
Efficacy was investigated in FIGHT-202 (NCT02924376), a multicenter open-label single-arm trial, in 107 patients with locally advanced unresectable or metastatic cholangiocarcinoma whose disease had progressed on or after at least one prior therapy and had an FGFR2 gene fusion or rearrangement. Patients received pemigatinib, 13.5 mg orally, once daily for 14 consecutive days, followed by 7 days off therapy.
The major efficacy outcome measures were overall response rate and duration of response determined by an independent review committee using RECIST 1.1. Among the 107 patients, the ORR was 36% (95% CI: 27%, 45%), including three complete responses. The median DOR was 9.1 months with responses lasting ≥ 6 months in 24 of the 38 (63%) responding patients and ≥ 12 months in 7 (18%) patients.
