The powerful FGFR kinase inhibitor Pemazyre EU is about to be approved for the introduction of Xinda Bio to China

In April 2020, the FDA approved Pemazyre for the treatment of patients with locally advanced or metastatic bile tube cancer who had previously received treatment, had FGFR2 fusion or rearm, and could not be surgically removed.
Pemazyre is approved under the Accelerated Approval Process and Priority Approval Process based on Total Mitigation Rate (ORR) and Mitigation Duration (DOR) data, and continued approval for the adaptation will depend on the validation and description of clinical benefits in the validation trial.
It's worth noting that Pemazyre is the first and only FDA-approved targeted drug for the treatment of bile tube cancer, which blocks the growth and spread of tumor cells by blocking FGFR2 in tumor cells.
Pemazyre has previously been granted orphan drug eligibility, breakthrough drug eligibility, and priority examination because bile tube cancer is a devastating cancer with serious and unsequal medical needs.
fda approval and positive review by CHMP of the European Union, based on data from theFIGHT-202 study.
the study was conducted in patients with localized advanced or metastatic bile tube cancer who had previously been treated to assess the efficacy and safety of Pemazyre.
The results of the study, recently presented at the 2019 congress of the European Society of Oncology (ESMO), showed that in patients carrying FGFR2 fusion or rearm (queue A), the average remission rate (ORR) of Pemazyre monotherapy was 36% (primary endpoint) and the medium duration of remission (DoR) was 9.1 months (secondary endpoint).
the study, adverse events were controllable and consistent with Pemazyre's mechanism of effect.
In patients treated with Pemazyre, the most common adverse reactions that occur in ≥20% of patients are high phosphorusemia and hypophosphateemia (electrolyte disorder), hair loss (baldness), diarrhea, nail toxicity, fatigue, taste disorder, nausea, constipation, stomatitis (pain or inflammation in the mouth), dry eye, dry mouth, loss of appetite, vomiting, joint pain, abdominal pain, back pain, and dry skin.
toxicity is also a risk to Pemazyre.
although bile tube cancer is considered a rare disease, the incidence has been rising for the past 30 years.
is very encouraging for patients who have previously received first-line chemotherapy or have limited options after surgery and have a high recurrence rate.
data from theFIGHT-202 study show that Pemazyre could offer new hope to these patients.
cholangiocarcinoma is a rare bile tube cancer that can be classified according to its anatomical origin: intra-hepatic bile tube cancer (iCCA) occurs in the intra-liver bile tube, and endo-hepatic bile tube cancer occurs in the outer bile tube of the liver.
patients with bile tube cancer are usually in the late or late stages of poor prognosticity at the time of diagnosis.
incidence of bile tube cancer varies from region to region, with rates in North America and Europe at 0.3-3.4/100,000.
FGFR2 fusion or rearm occurs almost exclusively in iCCA, observed in 10-16% of patients.
fibroblast growth factor (FGFR) plays an important role in tumor cell proliferation, survival, migration, and angiogenesy (the formation of new blood vessels).
fusion, rearfle, position and gene amplification in FGFR are closely related to the occurrence and development of various tumors.
Pemigatinib, the active pharmaceutical ingredient of Pemazyre, is a powerful, selective, oral small molecule inhibitor for FGFR isomers 1, 2, 3.
in preclinical studies, pemigatinib has been shown to have strong and selective pharmacological activity against cancer cells with FGFR gene changes.
currently, pemigatinib is being evaluated in a number of clinical studies to treat malignant tumors driven by FGFR gene mutations, including: bile tube cancer (Phase IIFIGHT-202, Phase IIIFIGHT-302), bladder cancer (Phase IIFIGHT-201), bone marrow proliferative tumor (8p11 MP) N, Phase IIFIGHT-203), Tumor-agnostic Cancer (tumor-agnostic, Phase IIFIGHT-207), bladder cancer (first-line therapy, Phase IIFIGHT-205), first-line treatment FGFR2 fusion or rearracing bile tube cancer (Phase IIIFIGHT-302).
December 2018, Cynda Bio and Incyte reached a strategic partnership and exclusive licensing agreement to advance the clinical development and commercialization of three drugs (itacitinib, parsaclisib, pemigatinib) for single-drug or combined treatments in Chinese mainland and Hong Kong, Macau and Taiwan.
under the terms of the agreement, Incyte will receive a $40 million down payment from Cyda Bio and a second $20 million cash payment after china first submitted its application for a new drug in 2019.
addition, Incyte will be eligible for potential development milestone payments of up to $129 million and potential business milestone payments of up to $202.5 million.
original origin: Incyte Announces Positive CHMP Opinion for Pemigatinib for The Treatment of Adults With With Previously Treated, Unresectable Local Advanced or Metastatic Cholangiocarcinoma With a Fibroblast Growth Factor Receptor 2 (FGFR2) or Fusion Rearrangement