RARE Daily

Europe Approves Incyte’s Pemazyre for Rare Cancer

March 30, 2021

Rare Daily Staff

The European Commission approved Incyte’s Pemazyre, a kinase inhibitor for the treatment of cholangiocarcinoma.

The approval was for adults with previously treated, locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed. The decision follows the positive opinion received from the European Medicines Agency’s Committee for Medicinal Products for Human Use in January 2021 recommending the conditional marketing authorization of Pemazyre.

“Pemazyre’s approval is a crucial milestone for patients with FGFR2 positive cholangiocarcinoma. It is the first new treatment option to be made available to these patients in the EU in over a decade and has demonstrated a high rate of durable responses in a setting where historically there has been no effective standard of care,” said Hervé Hoppenot, CEO, Incyte. “We now look forward to working with individual countries in Europe to ensure eligible patients can access this new treatment as soon as possible.”

Cholangiocarcinoma is a rare cancer that forms in bile ducts, which are slender tubes that carry the digestive fluid bile from the liver to gallbladder and small intestine. At diagnosis, a majority of patients with cholangiocarcinoma have advanced disease that is no longer treatable with surgery. Until now, their only option was a combination of chemotherapy drugs. FGFR2 fusions have been found in the tumors of approximately 9 percent to 14 percent of patients with cholangiocarcinoma. The incidence of cholangiocarcinoma in Europe ranges between 6,000 and 8,000.

Pemazyre is a tablet that works by blocking FGFR2 in tumor cells to prevent them from growing and spreading.

The EC decision is based on data from the FIGHT-202 study evaluating the safety and efficacy of Pemazyre in adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma with documented FGF/FGFR status. Interim results from FIGHT-202 demonstrated that in patients harboring FGFR2 fusions or rearrangements (n=108), Pemazyre monotherapy resulted in an overall response rate (ORR) of 37 percent (primary endpoint) and a median duration of response (DOR) of 8 months (secondary endpoint) based on an independent central radiographic review. Pemazyre was generally well tolerated. Warnings and precautions for Pemazyre include high and low levels of phosphate in the blood, vision or eye problems, blood creatinine increase, and for women who are pregnant, a risk of harm to the fetus.

“Historically, patients living with advanced cholangiocarcinoma have had very limited treatment options,” said Helen Morement, CEO, AMMF – The Cholangiocarcinoma Charity. “We are encouraged to see new, targeted therapies starting to be approved in Europe, giving hope to those in desperate need of alternatives.”

Photo: Hervé Hoppenot, CEO, Incyte

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