CLINICAL TRIALS PROFILE FOR PEMAZYRE

All Clinical Trials for Pemazyre

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT04088188 ↗	Gemcitabine and Cisplatin With Ivosidenib or Pemigatinib for the Treatment of Unresectable or Metastatic Cholangiocarcinoma	Recruiting	National Cancer Institute (NCI)	Phase 1	2021-01-25	This phase I trial studies the side effects and best dose of gemcitabine and cisplatin when given together with ivosidenib or pemigatinib in treating patients with cholangiocarcinoma that cannot be removed with surgery (unresectable) or has spread to other places in the body (metastatic). Chemotherapy drugs, such as gemcitabine and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Ivosidenib and pemigatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving gemcitabine and cisplatin with ivosidenib or pemigatinib may work better in treating patients with cholangiocarcinoma compared to gemcitabine and cisplatin alone.
NCT04088188 ↗	Gemcitabine and Cisplatin With Ivosidenib or Pemigatinib for the Treatment of Unresectable or Metastatic Cholangiocarcinoma	Recruiting	Academic and Community Cancer Research United	Phase 1	2021-01-25	This phase I trial studies the side effects and best dose of gemcitabine and cisplatin when given together with ivosidenib or pemigatinib in treating patients with cholangiocarcinoma that cannot be removed with surgery (unresectable) or has spread to other places in the body (metastatic). Chemotherapy drugs, such as gemcitabine and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Ivosidenib and pemigatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving gemcitabine and cisplatin with ivosidenib or pemigatinib may work better in treating patients with cholangiocarcinoma compared to gemcitabine and cisplatin alone.
NCT04659616 ↗	Pemigatinib After Chemotherapy for the Treatment of Newly Diagnosed Acute Myeloid Leukemia	Recruiting	Incyte Corporation	Phase 1	2021-01-14	This phase I trial identifies the best dose and clinical benefit of giving pemigatinib following standard induction chemotherapy in patients with newly diagnosed acute myeloid leukemia. Pemigatinib selectively inhibits FGFR (fibroblast growth factor receptor) activity, a receptor that may contribute to the growth of leukemia cells. The genetic changes responsible for activating the growth of leukemia cells can be unique to each patient and can change during the course of the disease. Chemotherapy drugs, such as cytarabine and daunorubicin work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.
NCT04659616 ↗	Pemigatinib After Chemotherapy for the Treatment of Newly Diagnosed Acute Myeloid Leukemia	Recruiting	Oregon Health and Science University	Phase 1	2021-01-14	This phase I trial identifies the best dose and clinical benefit of giving pemigatinib following standard induction chemotherapy in patients with newly diagnosed acute myeloid leukemia. Pemigatinib selectively inhibits FGFR (fibroblast growth factor receptor) activity, a receptor that may contribute to the growth of leukemia cells. The genetic changes responsible for activating the growth of leukemia cells can be unique to each patient and can change during the course of the disease. Chemotherapy drugs, such as cytarabine and daunorubicin work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.
NCT04659616 ↗	Pemigatinib After Chemotherapy for the Treatment of Newly Diagnosed Acute Myeloid Leukemia	Recruiting	OHSU Knight Cancer Institute	Phase 1	2021-01-14	This phase I trial identifies the best dose and clinical benefit of giving pemigatinib following standard induction chemotherapy in patients with newly diagnosed acute myeloid leukemia. Pemigatinib selectively inhibits FGFR (fibroblast growth factor receptor) activity, a receptor that may contribute to the growth of leukemia cells. The genetic changes responsible for activating the growth of leukemia cells can be unique to each patient and can change during the course of the disease. Chemotherapy drugs, such as cytarabine and daunorubicin work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

Clinical Trial Conditions for Pemazyre

Condition Name

Condition Name for Pemazyre
Intervention	Trials
Gastrointestinal Cancer	2
Advanced Cholangiocarcinoma	1
IDH1 NP_005887.2:p.R132H	1
Advanced Intrahepatic Cholangiocarcinoma	1

Condition MeSH

Condition MeSH for Pemazyre
Intervention	Trials
Gastrointestinal Neoplasms	2
Cholangiocarcinoma	2
Leukemia	1
Biliary Tract Neoplasms	1

Clinical Trial Locations for Pemazyre

Trials by Country

Trials by Country for Pemazyre
Location	Trials
United States	9
China	4

Trials by US State

Trials by US State for Pemazyre
Location	Trials
North Carolina	1
Nebraska	1
Minnesota	1
Georgia	1
Florida	1

Clinical Trial Progress for Pemazyre

Clinical Trial Phase

Clinical Trial Phase for Pemazyre
Clinical Trial Phase	Trials
Phase 2	5
Phase 1	2

Clinical Trial Status

Clinical Trial Status for Pemazyre
Clinical Trial Phase	Trials
Recruiting	6
Not yet recruiting	1

Clinical Trial Sponsors for Pemazyre

Sponsor Name

Sponsor Name for Pemazyre
Sponsor	Trials
Tianjin Medical University Cancer Institute and Hospital	2
Institut für Klinische Krebsforschung IKF GmbH at Krankenhaus Nordwest	1
National Cancer Institute (NCI)	1

Sponsor Type

Sponsor Type for Pemazyre
Sponsor	Trials
Other	8
Industry	2
NIH	1

PEMAZYRE: Clinical Trials, Market Analysis, and Projections

Introduction

PEMAZYRE, also known as pemigatinib, is a groundbreaking oral kinase inhibitor approved by the FDA in April 2020 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. Here, we delve into the clinical trials that led to its approval, the current market analysis, and future projections.

Clinical Trials Overview

The FDA approval of PEMAZYRE was based on evidence from a single-arm, open-label, Phase II trial known as Study FIGHT-202 (NCT02924376)[1][3][5].

Trial Design

• The trial enrolled 146 adult patients with bile duct cancer who had been treated previously with chemotherapy for their advanced cancer and whose tumors had an FGFR2 gene fusion or other rearrangement.
• Patients received PEMAZYRE in 21-day cycles, with 13.5 mg administered orally once daily for 14 consecutive days, followed by 7 days off therapy. Treatment continued until disease progression or unacceptable toxicity[1][3].

Efficacy Results

• The trial demonstrated a significant overall response rate (ORR) of 36%, with 38 out of 107 patients achieving partial or complete shrinkage of the tumor. Of these, 63% had a cancer shrinkage lasting 6 months or longer, and 18% had it lasting 12 months or longer[1][3].
• The median duration of response (DoR) was 9.1 months, with a 95% confidence interval of 6.0 to 14.5 months[1].

Market Analysis

Current Market Size

The global pemigatinib market, driven by PEMAZYRE, was valued at USD 95.11 million in 2024. This market is expected to grow significantly, reaching USD 258.76 million by 2032, with a compound annual growth rate (CAGR) of 9.83% during the forecast period of 2025 to 2032[2].

Market Segmentation

• The market is segmented by type (4.5 mg, 9 mg, and 13.5 mg), application (cholangiocarcinoma and others), dosage form (tablet and others), route of administration (oral and others), end-users (clinic, hospital, and others), and distribution channels (hospital pharmacy, retail pharmacy, and online pharmacy)[2].

Geographical Dominance

• North America is expected to dominate the pemigatinib market due to the presence of major key players, a well-developed healthcare sector, and advancements in treatment options. Asia-Pacific is also anticipated to show significant growth driven by increasing research and development activities and growing government support[2].

Market Trends

Precision Medicine

A significant trend in the pemigatinib market is the increasing emphasis on precision medicine, particularly in treating advanced cholangiocarcinoma. PEMAZYRE, as a selective inhibitor of FGFRs, has been specifically developed for patients with FGFR2 fusions or mutations, addressing an unmet need for targeted treatment options[2].

Companion Diagnostic

The FDA also approved Foundation Medicine’s FoundationOne CDx as a companion diagnostic for PEMAZYRE, which is a next-generation sequencing test. This test is crucial for identifying patients with FGFR2 gene fusions or other rearrangements, ensuring that only those who are likely to benefit from the treatment receive it[4][5].

Pricing and Revenue Projections

Pricing

PEMAZYRE is priced at $17,000 per treatment cycle, with an average treatment duration of around six months and eight or nine cycles. This translates to a cost per patient of between $136,000 and $153,000[5].

Revenue

RBC Capital Markets analysts predict that PEMAZYRE can generate $50 million in revenue in its first year. Given its exclusive rights in the U.S. and markets outside China, Hong Kong, Macau, and Taiwan, Incyte is well-positioned to capitalize on this market[5].

Future Projections and Expansion

Ongoing and Future Trials

PEMAZYRE is being evaluated in additional indications, including bladder cancer, solid tumors, and 8p11 myeloproliferative syndrome. The bladder cancer trial is expected to read out in the first half of 2021, although the timeline may be affected by the COVID-19 pandemic[5].

Market Growth Drivers

The market growth for PEMAZYRE is driven by several factors, including the increasing focus on precision medicine, the lack of effective treatments for cholangiocarcinoma, and the drug's favorable benefit-harm-uncertainty profile. As more patients are diagnosed with FGFR2-related cancers and as the drug expands into new indications, the market is expected to grow significantly[2][3].

Key Takeaways

• Clinical Efficacy: PEMAZYRE demonstrated a significant overall response rate and duration of response in patients with FGFR2 fusion or other rearrangements in cholangiocarcinoma.
• Market Size: The global pemigatinib market is projected to grow from USD 95.11 million in 2024 to USD 258.76 million by 2032.
• Precision Medicine: The drug's approval marks a significant milestone in precision medicine, targeting specific genetic mutations.
• Pricing and Revenue: With a list price of $17,000 per cycle, PEMAZYRE is expected to generate substantial revenue, particularly in North America and Asia-Pacific.
• Future Expansion: Ongoing and future trials in additional indications are expected to further expand the market for PEMAZYRE.

FAQs

What is PEMAZYRE used for?

PEMAZYRE is used 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[1][3][5].

How is PEMAZYRE administered?

PEMAZYRE is administered orally once daily at a dosage of 13.5 mg for 14 consecutive days, followed by 7 days off therapy, in 21-day cycles[1][3].

What is the overall response rate of PEMAZYRE in clinical trials?

In the clinical trials, 36% of patients treated with PEMAZYRE achieved partial or complete shrinkage of the tumor, with 63% of these patients experiencing cancer shrinkage lasting 6 months or longer[1][3].

What is the companion diagnostic for PEMAZYRE?

The FDA-approved companion diagnostic for PEMAZYRE is Foundation Medicine’s FoundationOne CDx, a next-generation sequencing test[4][5].

What are the future indications being explored for PEMAZYRE?

PEMAZYRE is being evaluated for additional indications, including bladder cancer, solid tumors, and 8p11 myeloproliferative syndrome[5].

How much does PEMAZYRE cost?

PEMAZYRE is priced at $17,000 per treatment cycle, with an average total cost per patient ranging from $136,000 to $153,000 for a typical treatment duration[5].

Sources

1. FDA: Drug Trials Snapshot: PEMAZYRE - FDA
2. Data Bridge Market Research: Global Pemigatinib Market Size, Share, and Trends Analysis Report
3. Health Canada: Summary Basis of Decision for Pemazyre
4. Value-Based Cancer Care: Pemazyre First FDA-Approved Therapy for Cholangiocarcinoma ...
5. FiercePharma: Incyte beefs up portfolio with FDA nod for rare bile duct cancer therapy Pemazyre