Pemazyre (pemigatinib)
An approved treatment for Cholangiocarcinoma.
The same compound appears under different names depending on the context. Here is how to identify Pemigatinib wherever you encounter it, plus the key facts at a glance.
- Generic name
- Pemigatinib
- Brand name
- Pemazyre
- Development code
- INCB054828
- Drug class
- Targeted small molecule (FGFR inhibitor)
- Manufacturer
- Incyte
- How it's taken
- Pemigatinib is taken orally at 13.
The first targeted therapy approved for cholangiocarcinoma with FGFR2 fusions. It is approved for adults whose cholangiocarcinoma has already been treated and cannot be removed by surgery or has spread, when an FDA-approved test finds an FGFR2 fusion or other rearrangement. This is an accelerated approval that FDA still lists as ongoing, so continued approval may depend on confirmatory trial results. A daily pill taken on a 2-weeks-on, 1-week-off schedule.
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Where Pemigatinib fits
First FGFR inhibitor approved for cholangiocarcinoma with FGFR2 fusions or rearrangements. Pemigatinib, futibatinib and, since September 23, 2026, lirafugratinib (Lyrfigtu) serve similar patient populations as sequential or alternative targeted options.
How Pemigatinib works
Pemigatinib blocks fibroblast growth factor receptors (FGFR), which are growth-promoting proteins commonly rearranged in cholangiocarcinoma. By blocking FGFR signaling, pemigatinib stops cancer cells from receiving the growth signals they depend on, causing them to stop dividing.
Mechanism: Selective FGFR1-3 inhibitor
Side effects and safety
The most common side effect is high blood phosphate, which can lead to calcium and phosphate deposits in soft tissues. Other common side effects include hair loss, diarrhea, nail changes, tiredness, taste changes, nausea, constipation, mouth sores, dry eyes and dry mouth. Pemazyre can also cause a serious eye problem called retinal pigment epithelial detachment, so you need an eye exam, including an OCT scan, before starting, every 2 months for the first 6 months, every 3 months after that, and right away if your vision changes. It can harm an unborn baby. Many side effects improve when the drug is paused or the dose is lowered.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Pemigatinib
Pemigatinib is taken orally at 13.5 mg once daily for 14 consecutive days, followed by 7 days off therapy (a 21-day cycle). This schedule is repeated until disease progression. Tablets can be taken with or without food.
Availability and cost
Only available as the brand-name product.
First FDA-approved targeted therapy for FGFR-altered cholangiocarcinoma. Orphan drug for a rare molecular subtype of a rare cancer.
Help paying for Pemazyre
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
Eligible patients with commercial prescription coverage may pay as little as $0; annual benefit maximum applies. Medicare, Medicaid, TRICARE and uninsured patients are not eligible.
For: private insurance · source - Free medicine program
Free PEMAZYRE for uninsured patients, Medicare Part D patients, or patients whose coverage was exhausted or denied, if they meet household income criteria.
For: no insurance, underinsured, Medicare · source - Bridge or quick-start supply
Free 30-day supply if coverage for an FDA-approved use is delayed; proof of the submitted insurance claim and delay is required.
The official page does not say who qualifies. Ask the program. · source - Insurance and case manager help
Help understanding insurance coverage, pharmacy outreach call at start, financial assistance options, and referrals to advocacy organizations.
The official page does not say who qualifies. Ask the program. · source
Good to know: PEMAZYRE is filled through Biologics by McKesson specialty pharmacy. Patients in Alternate Funding Programs are not eligible for free drug. Phone line Mon-Fri 8 AM-8 PM ET.
- From a charity · The Assistance FundBiliary Tract Cancer fundWaitlist
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
The foundation says: “WAITLIST — Accepting Waitlist Patients. TAF is currently accepting requests to join the enrollment waitlist for this program. Waitlists a…”
Clinical trial results
In the Phase 2 FIGHT-202 trial of 107 patients with FGFR2-rearranged cholangiocarcinoma, pemigatinib achieved a 35.5% response rate with durable responses. FDA granted accelerated approval on April 17, 2020, making it the first targeted therapy ever approved for cholangiocarcinoma.
Development history
Pemigatinib (INCB054828) was developed by Incyte Corporation as the first FGFR inhibitor approved for cholangiocarcinoma in 2020, opening a new treatment category for this previously hard-to-treat cancer.
Explore Cholangiocarcinoma trials
Other Cholangiocarcinoma treatments
Common questions about Pemigatinib
▸What is Pemigatinib (Pemazyre)?
The first targeted therapy approved for cholangiocarcinoma with FGFR2 fusions. It is approved for adults whose cholangiocarcinoma has already been treated and cannot be removed by surgery or has spread, when an FDA-approved test finds an FGFR2 fusion or other rearrangement. This is an accelerated approval that FDA still lists as ongoing, so continued approval may depend on confirmatory trial results. A daily pill taken on a 2-weeks-on, 1-week-off schedule.
▸How does Pemigatinib work?
Pemigatinib blocks fibroblast growth factor receptors (FGFR), which are growth-promoting proteins commonly rearranged in cholangiocarcinoma. By blocking FGFR signaling, pemigatinib stops cancer cells from receiving the growth signals they depend on, causing them to stop dividing.
▸What are the side effects of Pemigatinib?
The most common side effect is high blood phosphate, which can lead to calcium and phosphate deposits in soft tissues. Other common side effects include hair loss, diarrhea, nail changes, tiredness, taste changes, nausea, constipation, mouth sores, dry eyes and dry mouth. Pemazyre can also cause a serious eye problem called retinal pigment epithelial detachment, so you need an eye exam, including an OCT scan, before starting, every 2 months for the first 6 months, every 3 months after that, and right away if your vision changes. It can harm an unborn baby. Many side effects improve when the drug is paused or the dose is lowered.
▸How is Pemigatinib taken?
Pemigatinib is taken orally at 13.5 mg once daily for 14 consecutive days, followed by 7 days off therapy (a 21-day cycle). This schedule is repeated until disease progression. Tablets can be taken with or without food.
▸Is Pemigatinib FDA approved?
Yes, Pemigatinib (Pemazyre) is FDA approved (2020) for the treatment of Cholangiocarcinoma.
▸Why was pemigatinib historically significant for cholangiocarcinoma?
Pemigatinib (Pemazyre) was the first targeted therapy ever approved for cholangiocarcinoma when it received FDA accelerated approval in April 2020. It opened the era of precision medicine for this previously hard-to-treat cancer.
▸What is the dosing schedule for pemigatinib?
Pemigatinib follows a unique 2-weeks-on, 1-week-off schedule: 13.5 mg once daily for 14 consecutive days, then 7 days off (a 21-day cycle). This intermittent schedule helps manage side effects while maintaining efficacy.
▸What FGFR testing is required?
Tumor genomic testing must confirm FGFR2 fusions or rearrangements before starting pemigatinib. The drug only works in cancers driven by these specific genetic alterations.
▸What did the FIGHT-202 trial show?
The FIGHT-202 trial of 107 patients with FGFR2-rearranged cholangiocarcinoma showed a 35.5% response rate with durable responses, leading to FDA accelerated approval as the first targeted therapy for cholangiocarcinoma.
▸What are the main side effects?
High blood phosphate is the most characteristic effect. Other common side effects include hair loss, diarrhea, nail changes, tiredness, taste changes, nausea and dry eyes. Pemazyre can also cause a serious eye problem (retinal pigment epithelial detachment), so regular eye exams are required before and during treatment. Many side effects improve when the drug is paused or the dose reduced.
▸How does pemigatinib relate to futibatinib?
Both target FGFR2 fusions in cholangiocarcinoma and serve similar patient populations. Pemigatinib was approved first (2020) and binds FGFR reversibly, while futibatinib (2022) binds irreversibly. They can be considered sequential or alternative options.