PEMAZYRE
Pemazyre (pemigatinib) is a kinase inhibitor indicated for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma featuring a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement. This indication received accelerated approval based on overall response rate and duration of response, and continued approval may be contingent upon verification of clinical benefit in confirmatory trials. Additionally, the drug is indicated for adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with an FGFR1 rearrangement.
How PEMAZYRE Works
Pemigatinib is a small molecule kinase inhibitor that targets FGFR1, 2, and 3, and inhibits FGFR4 at significantly higher concentrations. The drug inhibits FGFR1-3 phosphorylation and signaling, which decreases cell viability in cancer cell lines with activating FGFR amplifications and fusions. By blocking constitutive FGFR signaling, pemigatinib disrupts the proliferation and survival of malignant cells.
Details
- Status
- Prescription
- First Approved
- 2020-04-17
- Routes
- ORAL
- Dosage Forms
- TABLET
PEMAZYRE Approval History
What PEMAZYRE Treats
2 indicationsPEMAZYRE is approved for 2 conditions since its original approval in 2020. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Cholangiocarcinoma
- Myeloid/Lymphoid Neoplasms
PEMAZYRE Competitors
Pro1 other drug also targets FGFR. Compare mechanisms, indications, and trial activity.
Competitors share the same molecular target (FGFR). Earlier expiry dates signal biosimilar/generic opportunities.
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Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
PEMAZYRE FDA Label Details
ProIndications & Usage
FDA Label (PDF)PEMAZYRE is a kinase inhibitor indicated: for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). For the treatment of adults with relapsed or refractory myeloid/lymphoid ...
PEMAZYRE Patents & Exclusivity
Patents (81 active)
Exclusivity
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.