New anti-cancer drugs: How effective is pemetinib/pemetinib?

U.S. Food and Drug Administration (FDA)Quasi-accelerated approvalpemetinib/pemetinib(Pemigatinib) is indicated for the treatment of adult patients with previously treated unresectable locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or other rearrangements as detected in an FDA-approved trial. The approval is based on the multicenter, open-label, single-arm trial of FIGHT-202.

Efficacy is based on107 patients with locally advanced unresectable or metastatic cholangiocarcinoma whose disease progressed during or after at least one prior line of therapy and who had FGFR2 gene fusions or rearrangements. Patients received 13.5 mg of pemetinib orally once daily for 14 days and then stopped treatment for 7 days. Safety was based on a total of 466 patients, 146 of whom had cholangiocarcinoma and received the recommended dose. Efficacy endpoints were overall response rate (ORR) and duration of response (DOR) as determined by an independent review committee using RECIST 1.1. The ORR was 36% (95% CI, 27-45). The median DOR was 9.1 months. Pemetinib has been proven to inhibit the growth and spread of tumors, causing disease stabilization or partial remission in some patients,The overall survival of patients in the treatment group was significantly longer than that in the placebo group.

The most common adverse reactions are hyperphosphatemia, alopecia, diarrhea, nail poisoning, fatigue, dysgeusia, nausea, constipation, stomatitis, dry eyes, dry mouth, decreased appetite, vomiting, joint pain, abdominal pain, hypophosphatemia, back pain, and dry skin. Ocular toxicity and hyperphosphatemia are important risks with pemetinib. The recommended dose is 13.5 mg orally once daily for 14 days, followed by 7 days off treatment in a 21-day cycle.