Incyte's Pemazyre becomes FDA's first and only approved unresectable, advanced cholangiocarcinoma treatment

The FDA has approved the United States’ first and only treatment for previously treated, unresectable locally advanced or metastatic cholangiocarcinoma, a rare form of bile duct cancer, with the marketing authorisation of Incyte’s Pemazyre (pemigatinib).
The kinase inhibitor is approved specifically in adult cholangiocarcinoma patients with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test.
Incyte scored the approval via the FDA’s Breakthrough Therapy and Orphan Drug designation pathways, sealing the decision after the drug demonstrated a 36% overall response as a monotherapy, meeting its main goal in clinical trials. Furthermore, the drug also met its secondary endpoint, displaying duration of response of 9.1 months.
Incyte CEO Hervé Hoppenot commented on the decision: “Our research into FGFR2 fusions or rearrangements in cholangiocarcinoma and the development of Pemazyre as the first targeted treatment option demonstrates our commitment to translating scientific discovery into solutions that can positively impact patients’ lives. We’re proud to bring Pemazyre to patients and will make this new treatment available immediately.”
Matt Fellows
- Login or register to post comments
- Printer-friendly version
Mission Statement
Pharmafile.com is a leading portal for the pharmaceutical industry, providing industry professionals with pharma news, pharma events, pharma service company listings and pharma jobs,
Site content is produced by our editorial team exclusively for Pharmafile.com and our industry newspaper Pharmafocus. Service company profiles and listings are taken from our pharmaceutical industry directory, Pharmafile, and presented in a unique Find and Compare format to ensure the most relevant matches
Related content
- FDA gives nod for Monjuvi combo for second-line relapsed or refractory diffuse large B-cell lymphoma
- Lilly and Incyte's resubmission of arthritis drug data fails to assuage FDA concerns
- Eli Lilly refutes FDA quality concerns at New Jersey facility producing COVID-19 drug
- FDA approves Venclexta combo in acute myeloid leukaemia, despite it missing main study goal
- FDA panel votes 9-1 in favour of approval for Mesoblast's Ryoncil in paediatric steroid-refractory acute graft versus host disease