Cinda Bio Partners Incyte's Powerful FGFR Kinase Inhibitor Pemazyre (pemigatinib) Was Approved By The US FDA!

- Mar 27, 2020-

Cinda Bio Partners Incyte recently announced that the US Food and Drug Administration (FDA) has approved Pemazyre (pemigatinib), which is a selective fibroblast growth factor receptor (FGFR) kinase inhibitor with indications of : It is used to treat patients with locally advanced or metastatic cholangiocarcinoma who have been previously treated, have FGFR2 fusion or rearrangement, and cannot be surgically removed. Pemazyre is approved under accelerated approval procedures and priority approval procedures based on total response rate (ORR) and duration of response (DOR) data. Continued approval for this indication will depend on the validation and clinical benefit of the confirmatory trial description. Currently, Pemazyre is also under review by the European Medicines Agency (EMA).


It is worth mentioning that Pemazyre is the first and only FDA approved drug for the treatment of cholangiocarcinoma, which can prevent the growth and spread of tumor cells by blocking FGFR2 in tumor cells. Because cholangiocarcinoma is a devastating cancer with serious unmet medical needs, Pemazyre has previously been granted orphan drug qualifications, breakthrough drug qualifications, and priority review qualifications.


This approval is based on data from the FIGHT-202 study. The study was conducted in patients with locally advanced or metastatic cholangiocarcinoma who had previously been treated to evaluate the efficacy and safety of Pemazyre. The results of this study were recently published at the European Society of Medical Oncology (ESMO) 2019 conference. The data showed that among patients with FGFR2 fusion or rearrangement (cohort A), the median follow-up was 15 months. The response rate (ORR) was 36% (primary endpoint), and the median duration of response (DoR) was 9.1 months (secondary endpoint).


In this study, adverse events were controllable and consistent with Pemazyre's mechanism of action. In patients receiving Pemazyre, the most common adverse reactions occurring in ≥20% of patients were hyperphosphatemia and hypophosphatemia (electrolyte disturbance), hair loss (alopecia areata), diarrhea, nail toxicity, fatigue, taste disturbance, nausea , Constipation, stomatitis (pain or inflammation in the mouth), dry eye, dry mouth, loss of appetite, vomiting, arthralgia, abdominal pain, back pain, and dry skin. Ocular toxicity is also a risk for Pemazyre.


Dr. Ghassan Abou-Alfa, MD, Memorial Sloan-Kettering Cancer Center, said: "Although cholangiocarcinoma is considered a rare disease, the incidence has been rising over the past 30 years. For those who have previously received first-line chemotherapy or surgery For a patient group with limited choices and high relapse rates, having a new targeted therapy is very encouraging and will bring new hope to patients. "


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Cholangiocarcinoma is a rare bile duct cancer, which can be classified according to its anatomical origin: intrahepatic cholangiocarcinoma (iCCA) occurs in intrahepatic bile ducts, and extrahepatic cholangiocarcinoma occurs in extrahepatic bile ducts. Patients with cholangiocarcinoma are usually in a late or late stage with a poor prognosis at the time of diagnosis. The incidence of cholangiocarcinoma varies by region, and the incidence in North America and Europe is 0.3-3.4 / 100000. FGFR2 fusion or rearrangement occurs almost exclusively in iCCA and is observed in 10-16% of patients.


Fibroblast growth factor receptor (FGFR) plays an important role in tumor cell proliferation, survival, migration and angiogenesis (neovascularization). Fusion, rearrangement, translocation and gene amplification in FGFR are closely related to the occurrence and development of various tumors.


Pemazyre's active pharmaceutical ingredient, pemigatinib, is a potent, selective, oral small molecule inhibitor against FGFR isomers 1, 2, and 3. In preclinical studies, pemigatinib has been shown to have potent and selective pharmacological activity against cancer cells that have undergone FGFR gene changes.

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pemigatinib (FGFR inhibitor, picture source: medchemexpress.cn)


Currently, pemigatinib is being evaluated in multiple clinical studies to treat malignant tumors driven by FGFR gene mutations, including: cholangiocarcinoma (stage II FIGHT-202, stage III FIGHT-302), bladder cancer (stage II FIGHT-201), bone marrow Proliferative tumor (8p11 MPN, stage II FIGHT-203), tumor agnostic cancer (tumor-agnostic, stage II FIGHT-207), bladder cancer (first-line treatment, stage II FIGHT-205), first-line treatment FGFR2 fusion or relapse Cholangiocarcinoma (stage III FIGHT-302).


In December 2018, Cinda Biotechnology and Incyte reached a strategic cooperation and exclusive licensing agreement to promote the clinical development of single drugs or combined treatment of three drugs (itacitinib, parsaclisib, pemigatinib) in mainland China and Hong Kong, Macau and Taiwan And commercialization. According to the terms of the cooperation agreement, Incyte will receive a down payment of US $ 40 million from Cinda Biotechnology and a second US $ 20 million cash payment after the first application for a new drug in China in 2019. In addition, Incyte will be eligible for up to $ 129 million in potential development milestone payments, and up to $ 202.5 million in potential business milestone payments.