FDA Approves First Targeted Treatment for Patients With Cholangiocarcinoma
The US Food and Drug Administration (FDA) granted accelerated approval to pemigatinib (Pemazyre), the first treatment approved for adults with certain types of previously treated, advanced cholangiocarcinoma.
“With [pemigatinib], we considered the observed efficacy results to be clinically meaningful and the overall risk to benefit assessment for patients with tumours harbouring FGFR2 gene fusions and other rearrangements to be favourable, particularly when we considered that these patients have no other good options following first line treatment with chemotherapy,” said Richard Pazdur, MD, FDA’s Center for Drug Evaluation and Research, Rockville, Maryland.
The approval was based on the results of a study of 107 patients with locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement who had received prior treatment. Patients received pemigatinib once a day for 14 consecutive days, followed by 7 days off, in 21-day cycles until the disease progressed or the patient experienced an unreasonable level of side effects. Patients were assessed every 8 weeks. The overall response rate was 36%, with 2.8% of patients having a complete response and 33% having a partial response. Among the 38 patients who had a response, 24 (63%) patients had a response lasting ≥6 months and 7 (18%) patients had a response lasting ≥12 months.
The most common adverse reactions occurring in ≥20% of patients who received pemigatinib are hyperphosphatemia and hypophosphatemia, alopecia, diarrhoea, nail toxicity, fatigue, dysgeusia, nausea, constipation, stomatitis, dry eye, dry mouth, decreased appetite, vomiting, joint pain, abdominal pain, back pain and dry skin. Ocular toxicity is also a risk with pemigatinib.
The FDA granted this application Priority Review and Breakthrough Therapy designation, which expedites the development and review of drugs that are intended to treat a serious condition, when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies. Pemigatinib received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. As a condition of the accelerated approval, the sponsor will complete and submit the results of a randomized trial demonstrating an improvement in progression-free survival or overall survival as a post-approval requirement.
SOURCE: US Food and Drug Administration