Regulatory approval

Published by the Food and Drug Administration.

The U.S. Food and Drug Administration granted accelerated approval to pemigatinib for the treatment of adult patients 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. The product label states that this indication is approved under accelerated approval based on overall response rate and duration of response and that continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

This is written in the approval document as:

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).

Citation

Incyte Corporation. Pemazyre (pemigatinib) [package insert]. U.S. Food and Drug Administration website. https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/213736s002lbl.pdf. Revised August 2022. Accessed October 30, 2024.

Therapeutic response

Precision oncology relationships for therapeutic response derived from this regulatory approval.

Type	Biomarker(s)	Cancer type	Therapy(ies)
Sensitivity (+)	FGFR2::v	Cholangiocarcinoma	Pemigatinib
Sensitivity (+)	FGFR2 rearrangements	Cholangiocarcinoma	Pemigatinib