With a coronavirus task force briefing unfolding at the White House late on April 17, an FDA eager to show its ongoing commitment to tackling other disease amid the pandemic, granted accelerated approval to Incyte Corp.'s Pemazyre (pemigatinib), the first treatment approved for adults with certain types of previously treated, advanced cholangiocarcinoma. It was one of multiple drugs the agency approved this year well ahead of their assigned PDUFA dates. Action on Pemazyre had been expected sometime around May 30.

The new drug, approved alongside a companion diagnostic, received a priority review following earlier designations as a breakthrough therapy and, in the U.S. and Europe, as an orphan drug. It remain under review by the EMA. As a condition of it continued U.S. approval, the Wilmington, Del.-based company will be required to complete and submit the results of a randomized trial demonstrating an improvement in progression-free survival.

"With Pemazyre, we considered the observed efficacy results to be clinically meaningful and the overall risk to benefit assessment for patients with tumors harboring FGFR2 gene fusions and other rearrangements to be favorable, particularly when we considered that these patients have no other good options following first line treatment with chemotherapy," said Richard Pazdur, director of the agency's Oncology Center of Excellence and acting director of its Office of Oncologic Diseases.

In tandem with the approval, Foundation Medicine Inc.'s genomic profiling assay, Foundationone CDx, gained the FDA's blessing for use in identifying patients with FGFR2 fusions and select rearrangements who may benefit from treatment with Pemazyre. It's now approved as the companion diagnostic test for 20 different therapies across multiple cancer types, the Cambridge, Mass.-based company said.

Cholangiocarcinoma is a rare form of cancer that forms in bile ducts. Though certain patients can be treated with surgery, a majority of patients at diagnosis have advanced disease, taking that option off the table, the FDA said. FGFR2 fusions are found in the tumors of 9% to 14% of patients with cholangiocarcinoma. Pemazyre works by blocking FGFR2 in tumor cells to prevent their growth and spread.

The drug was approved on the basis of data from the FIGHT-202 study, which evaluated pemigatinib as a treatment for patients with previously treated, locally advanced or metastatic cholangiocarcinoma. Study results, presented at the European Society for Medical Oncology 2019 Congress, demonstrated that in patients harboring FGFR2 fusions or rearrangements, pemigatinib monotherapy resulted in an overall response rate of 36%, and median duration of response of 7.5 months with a median follow-up of 15 months.

The most common adverse reactions to the drug, occurring in 20% or more of patients who received it during trial, include hyperphosphatemia and hypophosphatemia, alopecia, diarrhea, nail toxicity, fatigue, taste distortion, 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, the FDA said.

In January, the EMA validated an Incyte-filed MAA seeking approval. And in China, Suzhou-based Innovent Biologics Inc. said it dosed the first patient in a registrational trial in March. That work is part of a three-asset $391.5 million deal between Incyte and Innovent inked at the end of 2018 that also included itacitinib for graft-vs.-host disease and parsaclisib for non-Hodgkin's lymphoma. In contrast to Pemazyre's success, itacitinib fell flat in phase III test against acute GVHD.

Shares of Incyte (NASDAQ:INCY) fell 1% in early morning trading. Shares of Innovent (HKG:1801) climbed Monday, rising 5.85% to HK$TK (US$35.30).

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