MONDAY, April 20, 2020 (HealthDay News) -- Pemazyre (pemigatinib) has been granted accelerated approval for the treatment of advanced cholangiocarcinoma, the U.S. Food and Drug Administration announced Friday.
The drug approval is indicated for previously treated patients with locally advanced or metastatic cholangiocarcinoma and tumors with a fusion or other rearrangement of fibroblast growth factor receptor 2 (FGFR2). Until now, the standard initial treatment for these patients has been a combination of chemotherapy.
Approval was based on clinical trial data from 107 previously treated patients with locally advanced or metastatic cholangiocarcinoma and an FGFR2 fusion or rearrangement. Patients were treated in 21-day cycles that included treatment with Pemazyre once daily for 14 consecutive days followed by seven days off. They were treated until disease progression or until they experienced a high level of side effects. Based on scans every eight weeks, the researchers found an overall response rate of 36 percent; 2.8 percent of patients had a complete response, while 33 percent had a partial response. Among the patients with a response, 63 percent had a response lasting six months or longer and 18 percent had a response lasting one year or longer.
The most commonly reported adverse reactions (occurring in 20 percent or more of patients) were hyperphosphatemia and hypophosphatemia, alopecia, diarrhea, nail toxicity, fatigue, dysgeusia, nausea, constipation, stomatitis, dry eye, dry mouth, decreased appetite, vomiting, joint pain, abdominal pain, back pain, and dry skin. Patients taking Pemazyre are also at risk for ocular toxicity.
Approval was granted to Incyte Corporation.