CLINICAL TRIALS PROFILE FOR PLERIXAFOR

505(b)(2) Clinical Trials for PLERIXAFOR

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Indication	NCT00901225 ↗	Study of Plerixafor for Rescue of Poor Mobilizers in Autologous Stem Cell Transplant	Completed	Genzyme, a Sanofi Company	Phase 2	2009-05-01	Plerixafor, administered at a dose of 240 ug/kg, potentiates the effect of granulocyte colony-stimulating factor (G-CSF) to increase peripheral blood progenitor cells in both healthy volunteers and cancer patients. Furthermore, in cancer patients, cells collected via apheresis using Plerixafor and G-CSF have been successfully transplanted. In December 2008, Plerixafor received approval from the Food and Drug administration for use in combination with G-CSF to aid in mobilization of progenitor cells for apheresis. The proposed study is not designed to support approval of a new indication or change in the advertising for Plerixafor. The route of administration and dosage level are identical to that which is listed on the package insert. Although Plerixafor is not approved for patients with Hodgkins Lymphoma, there is no known or theoretic increased risk of the use of this drug in this patient population. The study hypothesis for this study is that patients with a circulating CD34+ count < 20 cells/ul after 5 days of mobilization with G-CSF alone will achieve > or equal to 2 X 10(6)CD34+ cells/kg within 3 days of apheresis after receiving Plerixafor with G-CSF.
New Indication	NCT00901225 ↗	Study of Plerixafor for Rescue of Poor Mobilizers in Autologous Stem Cell Transplant	Completed	Duke University	Phase 2	2009-05-01	Plerixafor, administered at a dose of 240 ug/kg, potentiates the effect of granulocyte colony-stimulating factor (G-CSF) to increase peripheral blood progenitor cells in both healthy volunteers and cancer patients. Furthermore, in cancer patients, cells collected via apheresis using Plerixafor and G-CSF have been successfully transplanted. In December 2008, Plerixafor received approval from the Food and Drug administration for use in combination with G-CSF to aid in mobilization of progenitor cells for apheresis. The proposed study is not designed to support approval of a new indication or change in the advertising for Plerixafor. The route of administration and dosage level are identical to that which is listed on the package insert. Although Plerixafor is not approved for patients with Hodgkins Lymphoma, there is no known or theoretic increased risk of the use of this drug in this patient population. The study hypothesis for this study is that patients with a circulating CD34+ count < 20 cells/ul after 5 days of mobilization with G-CSF alone will achieve > or equal to 2 X 10(6)CD34+ cells/kg within 3 days of apheresis after receiving Plerixafor with G-CSF.

All Clinical Trials for PLERIXAFOR

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00075335 ↗	AMD 3100 (Mozobil Plerixafor) to Mobilize Stem Cells for Donation	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 2	2004-01-01	Peripheral blood progenitor cells (PBPC) have become the preferred source of hematopoetic stem cells for allogeneic transplantation because of technical ease of collection and shorter time required for engraftment. Traditionally, granulocyte-colony stimulating factor (G-CSF) has been used to procure the peripheral blood stem cell graft. Although regimens using G-CSF usually succeed in collecting adequate numbers of PBPC from healthy donors, 5%-10% will mobilize stem cells poorly and may require multiple large volume apheresis or bone marrow harvesting. Although G-CSF is generally well tolerated in healthy donors, it may be associated with bone pain, headache, myalgia and rarely life threatening side effects like stroke, myocardial infarction and splenic rupture. AMD3100, is a bicyclam compound that inhibits the binding of stromal cell derived factor-1 (SDF-1) to its cognate receptor CXC- chemokine receptor 4 (CXCR4). CXCR4 is present on cluster of differentiation 34 (CD34)+ hematopoetic progenitor cells and its interaction with stromal cell derived factor 1 (SDF-1) plays a pivotal role in the homing of CD34+ cells in the bone marrow. Inhibition of the CXCR4-SDF1 axis by AMD3100 releases CD34+ cells into the circulation, which can then be collected easily by apheresis. Recently, a published report demonstrated that large numbers of CD34+ cells were rapidly mobilized in healthy volunteers following a single subcutaneous injection of AMD3100. Remarkably, the number of CD34+ cells collected by apheresis following a single injection of AMD3100 was comparable to the number of CD34+ cells collected from historical controls receiving 5 days of G-CSF prior to stem cell mobilization. In this study we will collect PBPCs following a single subcutaneous injection of AMD3100 from healthy donors who have previously had PBPC collected using standard G-CSF mobilization. The AMD3100 mobilized cells, G-CSF mobilized cells, and circulating cells prior to both AMD3100 and G-CSF mobilization will be analyzed in terms of cellular content and function of lymphocytes, natural killer (NK) cells, and antigen presenting cells. AMD3100 mobilized PBPC will be collected for the purpose of research studies and will not be used for therapeutic purposes.
NCT00082329 ↗	G-CSF and AMD3100 to Mobilize Stem Cells in Healthy Volunteers	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 2	2004-06-18	This 12-day study will test whether the combination of G-CSF (granulocyte-colony stimulating factor) and AMD3100 (Mozobil) is more efficient in mobilizing stem cells for collection than the use of G-CSF alone. Traditionally, the growth factor G-CSF has been given to stem cell donors to mobilize, or push, stem cells out of the bone marrow and into the blood circulation for collection for transplantation. Although a sufficient quantity of cells usually can be collected with G-CSF treatment, some donors do not respond well and may require multiple apheresis procedures (see below) to collect enough cells. Studies indicate that G-CSF used together with a drug called AMD3100 may be more effective in mobilizing stem cells for collection than G-CSF alone. The Food and Drug Administration has approved G-CSF for stem cell mobilization. AMD3100 is a new drug that also mobilizes stem cells in large numbers within a few hours. Normal healthy volunteers between 18 and 60 years of age may be eligible for this study.
NCT00082329 ↗	G-CSF and AMD3100 to Mobilize Stem Cells in Healthy Volunteers	Completed	Richard Childs, M.D.	Phase 2	2004-06-18	This 12-day study will test whether the combination of G-CSF (granulocyte-colony stimulating factor) and AMD3100 (Mozobil) is more efficient in mobilizing stem cells for collection than the use of G-CSF alone. Traditionally, the growth factor G-CSF has been given to stem cell donors to mobilize, or push, stem cells out of the bone marrow and into the blood circulation for collection for transplantation. Although a sufficient quantity of cells usually can be collected with G-CSF treatment, some donors do not respond well and may require multiple apheresis procedures (see below) to collect enough cells. Studies indicate that G-CSF used together with a drug called AMD3100 may be more effective in mobilizing stem cells for collection than G-CSF alone. The Food and Drug Administration has approved G-CSF for stem cell mobilization. AMD3100 is a new drug that also mobilizes stem cells in large numbers within a few hours. Normal healthy volunteers between 18 and 60 years of age may be eligible for this study.

Clinical Trial Conditions for PLERIXAFOR

Condition Name

Condition Name for PLERIXAFOR
Intervention	Trials
Multiple Myeloma	35
Lymphoma	11
Non-Hodgkin's Lymphoma	11

Condition MeSH

Condition MeSH for PLERIXAFOR
Intervention	Trials
Multiple Myeloma	44
Lymphoma, Non-Hodgkin	36
Neoplasms, Plasma Cell	35

Clinical Trial Locations for PLERIXAFOR

Trials by Country

Trials by Country for PLERIXAFOR
Location	Trials
United States	232
Spain	17
Canada	11
Italy	8
France	7

Trials by US State

Trials by US State for PLERIXAFOR
Location	Trials
California	24
Missouri	21
Maryland	18
New York	16
Texas	15

Clinical Trial Progress for PLERIXAFOR

Clinical Trial Phase

Clinical Trial Phase for PLERIXAFOR
Clinical Trial Phase	Trials
PHASE3	2
PHASE2	2
PHASE1	5

Clinical Trial Status

Clinical Trial Status for PLERIXAFOR
Clinical Trial Phase	Trials
Completed	82
Recruiting	31
Terminated	23

Clinical Trial Sponsors for PLERIXAFOR

Sponsor Name

Sponsor Name for PLERIXAFOR
Sponsor	Trials
Genzyme, a Sanofi Company	45
National Cancer Institute (NCI)	16
Washington University School of Medicine	13

Sponsor Type

Sponsor Type for PLERIXAFOR
Sponsor	Trials
Other	217
Industry	90
NIH	33

Clinical Trials Update, Market Analysis, and Projection for Plerixafor

Last updated: October 28, 2025

Introduction

Plerixafor, marketed as Mozobil, is a CXCR4 chemokine receptor antagonist developed primarily to enhance stem cell mobilization for autologous transplantation in patients with non-Hodgkin's lymphoma and multiple myeloma. Originally approved by the U.S. Food and Drug Administration (FDA) in 2008, its utility has expanded with ongoing clinical trials exploring new indications. As the global hematology and oncology markets evolve, understanding the trajectory of Plerixafor’s clinical development, competitive positioning, and future market opportunities is crucial for stakeholders.

Clinical Trials Landscape

Current Clinical Trials and Developments

Plerixafor’s clinical development trajectory remains active, with several trials assessing its efficacy beyond hematopoietic stem cell mobilization. Recent updates from ClinicalTrials.gov indicate:

• Oncology & Hematology: Numerous ongoing Phase II and III trials focus on combining Plerixafor with other agents for treating multiple myeloma, lymphoma, and acute myeloid leukemia (AML). For example, a 2022 study (NCT0482252) explores Plerixafor combined with chemotherapy agents to improve remission rates in AML patients.

• Solid Tumors: Early-phase trials investigate CXCR4 inhibition as an adjunct to immunotherapy in solid tumors. Notably, NCT04558136 is evaluating Plerixafor in combination with pembrolizumab for metastatic pancreatic cancer.

• Stem Cell Mobilization in Non-Malignant Diseases: Trials continue in mobilizing stem cells for regenerative medicine indications, including cardiovascular diseases and neurodegenerative conditions.

Regulatory and Approval Milestones

Recent approvals and regulatory assessments reflect Plerixafor’s expanding therapeutic profile:

• Expanded Indications: The FDA’s approval of Plerixafor for multiple myeloma and non-Hodgkin’s lymphoma in 2016 has cemented its role in hematopoietic stem cell transplantation. An upcoming submission in Europe aims to broaden its use in multiple myeloma.

• Orphan Drug Designations: Some trials targeting rare hematological malignancies benefit from orphan status, ensuring market exclusivity and supportive regulatory pathways.

Safety and Efficacy Data

Meta-analyses indicate Plerixafor’s acceptable safety profile, with common adverse effects including injection site reactions, diarrhea, and transient leukocytosis. Efficacy data from Phase III trials confirm its superior mobilization capabilities over G-CSF alone, with an increase in CD34+ cell yield.

Market Analysis

Market Size and Growth Drivers

The global hematopoietic stem cell mobilization market was valued at approximately USD 300 million in 2021 and is projected to grow at a CAGR of 8-10% over the next five years [1]. The primary drivers include:

• Rising incidence of hematological malignancies.
• Increased adoption of autologous stem cell transplantation.
• Expansion of Plerixafor's indications and off-label uses.

Competitive Landscape

Throughout its lifecycle, Plerixafor has faced competition from other mobilization agents such as G-CSF monotherapy. However, its unique mechanism offers significant advantages, notably:

• Enhanced stem cell yield in poor mobilizers.
• Reduced mobilization failure rates, improving transplant success.

Emerging competitors include newer CXCR4 antagonists and novel mobilizing agents under clinical evaluation—such as BL-8040 (Motixafortide)—which demonstrate comparable or superior efficacy.

Market Penetration

In North America and Europe, Plerixafor enjoys widespread adoption in transplantation centers. Market penetration remains robust due to reimbursement policies and clinician familiarity. However, high costs (approx. USD 10,000 per dose) pose barriers in some regions, potentially limiting access in emerging markets.

Pricing and Reimbursement Trends

Pricing strategies are influenced by:

• Cost-effectiveness: Analyses indicate that Plerixafor reduces the need for remobilization and hospitalization, offsetting medication costs.
• Reimbursement policies: Payers increasingly favor pharmacoeconomic evaluations, supporting coverage approvals.

Adaptive reimbursement frameworks and participation in value-based agreements aid expansion into developing economies.

Market Projection

Forecasted Growth Trajectory

Based on current data, Plerixafor’s global market is expected to grow at a CAGR of approximately 9% through 2030, reaching around USD 600-700 million by the end of the decade [1]. Key factors influencing this include:

• Expansion of indications, particularly in chemo-mobilization protocols and emerging cellular therapies.
• Increased utilization in clinical trials for novel combinations.
• Emerging markets’ adoption driven by increasing healthcare infrastructure.

Potential Upside Opportunities

• Combination therapies: Trials combining Plerixafor with monoclonal antibodies or CAR-T therapies could unlock new markets.
• Regulatory approvals for non-hematologic indications: Demonstrable efficacy in solid tumors and regenerative medicine could diversify revenue streams.
• Biosimilar developments: Although early-stage, biosimilar versions could reduce costs and facilitate broader access.

Challenges and Risks

• Pricing pressures and reimbursement hurdles.
• Development of superior competitors.
• Regulatory setbacks in new indications.
• Market saturation in established regions.

Key Takeaways

• Plerixafor remains a cornerstone agent for stem cell mobilization, with ongoing clinical trials expanding its utility.
• The global market is projected to double by 2030, driven by clinical expansion and increasing hematological disease prevalence.
• Competitive dynamics threaten market share; maintaining differentiation through proven efficacy and safety is essential.
• Cost and reimbursement considerations will influence penetration in emerging markets.
• Strategic collaborations and indication expansions present significant growth opportunities.

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FAQs

1. What are the primary clinical indications for Plerixafor today?
Plerixafor is primarily indicated for autologous hematopoietic stem cell mobilization in patients with multiple myeloma and non-Hodgkin's lymphoma who are poor mobilizers, often used in conjunction with G-CSF.

2. Are there upcoming regulatory approvals that could expand Plerixafor’s market?
Yes. Trials exploring its use in solid tumors and regenerative medicine could lead to regulatory submissions, potentially broadening its approval landscape.

3. How does Plerixafor compare to other mobilization agents?
Plerixafor offers superior mobilization efficacy in poor mobilizers compared to G-CSF alone and reduces mobilization failure rates, making it a preferred agent in high-risk groups.

4. What are the main challenges facing Plerixafor’s market growth?
High costs, competition from emerging agents, and reimbursement hurdles, especially in low-resource settings, pose significant challenges.

5. How might Plerixafor fit into future cellular therapies?
Its ability to mobilize stem cells efficiently makes it a valuable agent for upcoming cellular therapies, including CAR-T cells and regenerative applications, presenting multiple growth avenues.

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Sources

[1] Market Research Future, "Hematopoietic Stem Cell Mobilization Market," 2022.