FDA Approves First Targeted Treatment for Patients with Chol
U.S. Food and Drug Administration granted accelerated approval to Pemazyre (pemigatinib), the first treatment approved for adults with certain types of previously treated, advanced cholangiocarcinoma.

Cholangiocarcinoma is a rare form of cancer that forms in bile ducts, which are slender tubes that carry the digestive fluid bile from the liver to gallbladder and small intestine. This approval is for patients with cholangiocarcinoma that is locally advanced or metastatic and who have tumors that have a fusion or other rearrangement of a gene called fibroblast growth factor receptor 2 (FGFR2).

For these patients, there have been no FDA-approved therapies; a combination of chemotherapy drugs has been the standard initial treatment. 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 to prevent them from growing and spreading.

Pemazyre’s approval was based on the results of a clinical trial that enrolled 107 patients with locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement who had received prior treatment. During the clinical trial, patients received Pemazyre 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. To assess how well Pemazyre was working during the trial, patients were scanned every eight weeks. The trial used established criteria to measure how many patients experienced a complete or partial shrinkage of their tumors during treatment (overall response rate). 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 patients (63%) had a response lasting 6 months or longer and 7 patients (18%) had a response lasting 12 months or longer.

The most common adverse reactions occurring in 20% or more of patients who received Pemazyre are hyperphosphatemia and hypophosphatemia (electrolyte disorders), alopecia (spot baldness), diarrhea, nail toxicity, fatigue, dysgeusia (taste distortion), nausea, constipation, stomatitis (sore or inflammation inside the mouth), dry eye, dry mouth, decreased appetite, vomiting, joint pain, abdominal pain, back pain, and dry skin. Ocular (eye) toxicity is also a risk of Pemazyre.

The FDA granted approval of Pemazyre to Incyte Corporation.
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