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Pemazyre
Published
Product name
Pemazyre
Active ingredient
Pemigatinib
Submission type
New chemical entity
Decision
Approved for provisional registration
Decision date
Registration date
What this medicine was approved for
Pemazyre (pemigatinib) was approved for the following therapeutic use:
Pemigatinib has provisional approval in Australia for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that has progressed after at least one prior line of systemic therapy. The decision to approve this indication has been made on the basis of overall response rate (ORR) and duration of response (DOR). Continued approval of this indication depends on verification and description of benefit in confirmatory trial(s).
How this medicine works
Pemigatinib is a small molecule kinase inhibitor of fibroblast growth factor receptor (FGFR)1, 2 and 3 with half maximal inhibitory concentration (IC50) values of less than 2 nM. Pemigatinib also inhibited FGFR4 in vitro at a concentration approximately 100 times higher than those that inhibit FGFR1, 2, and 3. It inhibits FGFR1, 2 and 3 phosphorylation and signalling and decreases cell viability in cancer cell lines with activating FGFR genetic alterations, including point mutations, amplifications, and fusions or rearrangements that resulted in constitutive activation of FGFR signalling. FGFR2 fusions or rearrangements are strong oncogenic drivers and are the most common FGFR alteration occurring, almost exclusively, in 10 to 16% of intrahepatic cholangiocarcinoma. Constitutive FGFR signalling can support the proliferation and survival of malignant cells. Pemigatinib exhibited anti-tumour activity in mouse xenograft models of human tumours with FGFR1, FGFR2, or FGFR3 alterations resulting in constitutive FGFR activation including a patient-derived xenograft model of cholangiocarcinoma that expressed an oncogenic FGFR2 transformer-2 beta homolog (TRA2b) fusion protein.
Why the TGA approved or did not approve this medicine
The decision was based on quality (chemistry and manufacturing), nonclinical (pharmacology and toxicology), clinical (pharmacology, safety and efficacy) and risk management plan information submitted by the sponsor. The benefit-risk profile of Pemazyre was considered favourable for the therapeutic use approved.