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Pemazyre (PEMIGATINIB)
Pemazyre blocks the activity of the FGFR1 protein, which is involved in the growth and spread of cancer cells.
Pemazyre (pemigatinib) is a small molecule kinase inhibitor developed by Incyte Corp, targeting fibroblast growth factor receptor 1 (FGFR1). It was FDA-approved in 2020 for the treatment of biliary tract cancer, FGFR1 fusion gene-positive myeloid/lymphoid neoplasm, and malignant neoplasm of the common bile duct. Pemazyre is a patented medication with no generic manufacturers available. Key safety considerations include potential liver damage and gastrointestinal side effects. It is a targeted therapy that specifically blocks the activity of the FGFR1 protein, which is involved in the growth and spread of cancer cells.
At a glance
| Generic name | PEMIGATINIB |
|---|---|
| Sponsor | Incyte Corp |
| Drug class | Kinase Inhibitor [EPC] |
| Target | Fibroblast growth factor receptor 1 |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
| First approval | 2020 |
| Annual revenue | 200 |
Mechanism of action
Pemigatinib is small molecule kinase inhibitor that targets FGFR1, and with IC50 values of less than nM. Pemigatinib also inhibited FGFR4 in vitro at concentration approximately 100 times higher than those that inhibit FGFR1, 2, and 3. Pemigatinib inhibited FGFR1-3 phosphorylation and signaling and decreased cell viability in cancer cell lines with activating FGFR amplifications and fusions that resulted in constitutive activation of FGFR signaling. Constitutive FGFR signaling can support the proliferation and survival of malignant cells. Pemigatinib exhibited anti-tumor activity in mouse xenograft models of human tumors with FGFR1, FGFR2, or FGFR3 alterations resulting in constitutive FGFR activation including patient-derived xenograft model of cholangiocarcinoma that expressed an oncogenic FGFR2-Transformer-2 beta homolog (TRA2b) fusion protein.
Approved indications
- Biliary tract cancer
- FGFR1 fusion genepositive myeloid/lymphoid neoplasm
- Malignant neoplasm of common bile duct (& [carcinoma])
Common side effects
- hyperphosphatemia
- decreased appetite
- hypophosphatemia
- dehydration
Key clinical trials
- A Phase II Study of Pemigatinib Plus Durvalumab in Previously Treated Advanced Intrahepatic Cholangiocarcinoma Patients With FGFR-2 Fusion or Rearrangement (PHASE2)
- PH 2 Pemigatinib in SDH-deficient GIST (PHASE2)
- A Phase II Nationwide, Fully Decentralized, Telemedicine Study of Pemigatinib in Adult Patients With Advanced or Metastatic Pancreatic Cancer With FGFR Genetic Alterations (PHASE2)
- Pemigatinib + Afatinib in Advanced Refractory Solid Tumors (PHASE1)
- Phase I/II Trial of Pemigatinib in Combination With Atezolizumab and Bevacizumab for Treatment of Advanced Cholangiocarcinoma With FGFR2 Fusion (PHASE1,PHASE2)
- Study to Evaluate the Efficacy and Safety of Pemigatinib in Participants With Previously Treated Glioblastoma or Other Primary Central Nervous System Tumors Harboring Activating FGFR1-3 Alterations (PHASE2)
- Pemigatinib After Chemotherapy for the Treatment of Newly Diagnosed Acute Myeloid Leukemia (PHASE1)
- Safety and Efficacy of Retifanlimab (INCMGA00012) Alone or in Combination With Other Therapies in Participants With Advanced or Metastatic Endometrial Cancer Who Have Progressed on or After Platinum-based Chemotherapy. (PHASE2)
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
| SEC EDGAR | Revenue + earnings |