FDA批准佩米替尼(pemigatinib)作为首个治疗既往接受过治疗、无法切除的局部晚期或转移性胆管癌成人患者的靶向治疗药物

FDA approves pemigatinib as the first targeted therapy for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma

‌ April 17, 2020‌ Wilmington, Del., Business Wire.

Incyte announced that the U.S. Food and Drug Administration (FDA) has approved the marketing of this kinase inhibitor for the treatment of adult patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma confirmed by FDA-approved testing to have fibroblast growth factor receptor 2 (FGFR2) fusions or other rearrangements. Pemigatinib is the first and only drug approved by the FDA for this indication. Its approval is based on the accelerated approval pathway and is based on overall response rate (ORR) and duration of response (DOR). Subsequent approval may be contingent on verification and description of clinical benefit in confirmatory trials.

Hervé Hoppenot, CEO of Incyte, said: "Our series of studies on FGFR2 fusion or rearrangement in cholangiocarcinoma and the development of pemigatinib as the first targeted treatment option are the best examples of how we can transform scientific discoveries into real positive impacts on patients' lives. With pemigatinib With the successful approval, we have brought new hope to patients for a better treatment, and immediately included it in the new round of clinical treatment options.”

The FDA approved it based on preliminary research data from FIGHT-202, a multi-center, open-label, single-arm clinical study on the therapeutic effect of pemigatinib in adult patients with cholangiocarcinoma. As a monotherapy for patients with FGFR2 fusions or rearrangements, preliminary results from clinical trials of pemetinib show that it can achieve an overall response rate of 36%, with a median duration of response of 9.1 months.

Pemigatinib Precautions

Eye problems (such as dry or inflamed eyes, corneal inflammation, increased tearing, and retinal disorders); increased phosphate levels in the blood; and pregnant women are at risk of harm to the fetus or leading to miscarriage.

The FDA granted pemigatinib Breakthrough Therapy designation for the treatment of patients with previously treated advanced/metastatic or unresectable FGFR2 translocation cholangiocarcinoma. The FDA also granted pemigatinib orphan drug designation for the treatment of cholangiocarcinoma, and its New Drug Application (NDA) was reviewed through the FDA's priority review process.

Ghassan Experts from the Abou-Alfa Sloan-Kettering Cancer Center believe: "Although cholangiocarcinoma is considered a rare malignant tumor, its incidence has continued to increase in the past three decades, accounting for approximately more than 20% of pancreatic cancers. For patients with limited options after first-line chemotherapy or surgery, new targeted treatment options make Encouragingly, recurrence rates remain high in these patients. ”

Overview of Cholangiocarcinoma

Cholangiocarcinoma is a rare cancer that forms in the bile ducts and is divided into two categories based on its anatomical origin: intrahepatic cholangiocarcinoma (iCCA), which occurs in the bile ducts within the liver, and extrahepatic cholangiocarcinoma, which occurs in the bile ducts outside the liver. Patients with cholangiocarcinoma are often diagnosed at an advanced stage or when the prognosis is poor. In North America and Europe, the incidence of cholangiocarcinoma varies widely between regions, ranging from 0.3 to 3.4 cases per 100,000 people. FGFR2 fusion or rearrangement is almost exclusively seen in iCCA, accounting for approximately 10%-16% of patients. FGFR plays an important role in tumor cell proliferation and survival, migration, and angiogenesis (new blood vessel formation). Activating fusion, rearrangement, translocation and gene amplification of FGFR are closely related to the development of various cancers.

Stacie Lindsey, President of the Cholangiocarcinoma Foundation, said: "The approval of pemigatinib provides patients with an exciting new treatment option and will bring hope to patients who often face a difficult diagnosis journey and poor prognosis."

Incyte is committed to supporting patients and removing barriers to accessing medicines. Eligible patients prescribed pemigatinib in the United States are supported by the IncyteCARES (Connecting Access, Reimbursement, Education and Support) program, a comprehensive program that provides ongoing education and resources, including financial assistance, to eligible patients. For more information, visit pemigatinib.com.

The FDA is also expected to approve FoundationOne®CDx as a companion diagnostic to pemigatinib. With in-depth analysis of the entire genome, FoundationOne CDx provides comprehensive genomic interpretation for companion diagnostics in all solid tumors. If approved, the companion diagnostic will help identify patients with FGFR2 fusions or rearrangements who may be candidates for treatment with pemigatinib.