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.
NCT00103610 ↗	Mobilization of Stem Cells With AMD3100 (Plerixafor) in Non-Hodgkin's Lymphoma Patients	Completed	Genzyme, a Sanofi Company	Phase 3	2005-01-01	The purpose of this study is to determine whether the combination of AMD3100 (plerixafor) and granulocyte colony-stimulating factor (G-CSF or generic name filgrastim) is better than G-CSF alone to mobilize and collect the optimal number of stem cells in non-Hodgkin's lymphoma patients for autologous transplantation.

Clinical Trial Conditions for PLERIXAFOR

Condition Name

Condition Name for PLERIXAFOR
Intervention	Trials
Multiple Myeloma	35
Non-Hodgkin's Lymphoma	11
Lymphoma	11
Acute Myeloid Leukemia	10

Condition MeSH

Condition MeSH for PLERIXAFOR
Intervention	Trials
Multiple Myeloma	44
Lymphoma, Non-Hodgkin	36
Neoplasms, Plasma Cell	35
Lymphoma	33

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	6

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	218
Industry	90
NIH	33

Plerixafor: Clinical Trials, Market Dynamics, and Future Outlook

Last updated: February 19, 2026

Plerixafor, a CXCR4 antagonist, is an established agent primarily used in hematopoietic stem cell transplantation. Recent clinical trial data and ongoing research indicate potential for expanded indications, influencing its market trajectory. Key developments center on its efficacy in combination therapies and its role in managing specific oncological and hematological conditions.

What is the Current Status of Plerixafor's Clinical Development?

Plerixafor is approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for use in mobilizing hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with non-Hodgkin's lymphoma (NHL) and multiple myeloma (MM) [1, 2].

Recent and ongoing clinical trials are exploring plerixafor in several areas:

• Oncological Indications:
  • Acute Myeloid Leukemia (AML): Trials are investigating plerixafor's efficacy in combination with chemotherapy for AML induction and mobilization of leukemic stem cells for treatment [3]. Some studies aim to assess its role in reducing minimal residual disease (MRD) post-transplant.
  • Myelodysplastic Syndromes (MDS): Research is ongoing to evaluate plerixafor as a component of treatment regimens for MDS, particularly in specific genetic subtypes where CXCR4 signaling is implicated [4].
  • Solid Tumors: Early-phase studies are examining plerixafor's potential to enhance the efficacy of chemotherapy or immunotherapy in certain solid tumors, such as pancreatic cancer, by disrupting the tumor microenvironment and improving drug penetration [5].
• Hematological Indications (Beyond Stem Cell Mobilization):
  • Sickle Cell Disease (SCD): Plerixafor is being studied for its ability to induce a relative increase in fetal hemoglobin (HbF) production. By inhibiting the CXCR4 pathway, it may stimulate the production of gamma-globin, a component of HbF, potentially alleviating vaso-occlusive crises [6, 7]. Clinical trials have shown promising results in reducing pain crises.
  • Aplastic Anemia: Research is exploring plerixafor's potential to improve engraftment and outcomes in patients undergoing allogeneic hematopoietic stem cell transplantation for aplastic anemia [8].
• Other Investigational Uses:
  • Graft-versus-Host Disease (GvHD) Prophylaxis: Preliminary investigations are assessing plerixafor's role in modulating immune responses to potentially reduce the incidence or severity of acute and chronic GvHD following allogeneic transplantation [9].

Table 1: Key Plerixafor Clinical Trials and Indications

Indication	Phase	Status	Primary Endpoint(s)	Key Findings/Notes
AML (Stem Cell Mobilization)	III	Completed	Stem cell yield, engraftment time	Established efficacy in enhancing stem cell collection for autologous transplant.
AML (AML Stem Cell Mobilization)	II	Ongoing	Reduction in MRD, overall survival	Investigating plerixafor's role in clearing residual leukemic cells.
Sickle Cell Disease (Pain Crises)	III	Ongoing	Frequency of vaso-occlusive crises, time to symptom resolution	Promising results in reducing pain crisis frequency and duration.
Pancreatic Cancer (TME modulation)	II	Ongoing	Tumor response rates, progression-free survival, drug penetration into tumor microenvironment	Aiming to improve efficacy of chemotherapy by altering tumor microenvironment.
MDS (Treatment Adjunct)	I/II	Ongoing	Hematological response, cytogenetic response	Exploring efficacy in specific MDS subtypes linked to CXCR4 signaling.
Aplastic Anemia (Engraftment)	II	Ongoing	Hematopoietic engraftment rates, overall survival	Assessing plerixafor's impact on outcomes in allogeneic SCT for aplastic anemia.

What is the Current Market Landscape for Plerixafor?

Plerixafor, marketed as Mozobil by Sanofi (following its acquisition of Genzyme), holds a significant position in the stem cell transplantation market. Its primary indication for stem cell mobilization in NHL and MM has established a consistent revenue stream.

• Market Size and Growth: The global market for stem cell mobilization agents is driven by the increasing incidence of hematological malignancies and advancements in transplantation procedures. While precise market figures for plerixafor alone are proprietary, the broader market for stem cell transplantation therapies is projected to grow.
• Competitive Landscape:
  • Current Competition: In its approved indication, plerixafor primarily competes with granulocyte-colony stimulating factor (G-CSF) alone or in combination with plerixafor. G-CSF is a more established and generally lower-cost alternative, but plerixafor offers enhanced stem cell yield and faster engraftment in specific patient populations.
  • Emerging Competition: The development of novel agents targeting CXCR4 or alternative pathways for stem cell mobilization and for the treatment of diseases like SCD and AML could introduce new competitive pressures in the future.
• Pricing and Reimbursement: Plerixafor is a high-cost therapy, with pricing reflecting its specialized use and clinical value. Reimbursement policies by payers are critical for market access and adoption. The established reimbursement for its approved indications supports its current market position.
• Geographic Distribution: North America and Europe represent the largest markets for plerixafor due to the established healthcare infrastructure, higher rates of stem cell transplantation, and favorable reimbursement landscapes. Asia-Pacific is emerging as a significant growth region.

Table 2: Market Factors for Plerixafor

Factor	Description	Impact on Plerixafor
Approved Indications	Autologous stem cell mobilization for NHL and MM.	Provides a stable, established market base and revenue stream.
Pipeline Expansion	Ongoing trials in AML, SCD, MDS, and solid tumors.	Potential to significantly expand market reach and revenue if new indications are approved.
Competition	G-CSF (primary), potential novel CXCR4 inhibitors or alternative pathway agents.	G-CSF limits broader adoption for mobilization in all eligible patients due to cost. Novel agents could challenge existing or future indications.
Pricing	High-cost specialty drug.	Requires strong clinical justification and favorable reimbursement for market access. May limit use in resource-constrained settings.
Reimbursement	Established coverage for approved indications in major markets.	Crucial for market penetration. Changes in payer policies could impact accessibility.
Healthcare Trends	Increasing cancer incidence, growing adoption of SCT, interest in disease-modifying therapies for chronic conditions.	Favorable trends for demand in approved indications and supportive for potential new indications.
Manufacturing & Supply	Complex manufacturing process, reliance on specialized facilities.	Potential for supply chain vulnerabilities or cost pressures if demand increases significantly or manufacturing issues arise.

What are the Market Projections and Future Outlook for Plerixafor?

The future market performance of plerixafor is contingent on several factors, primarily the success of its ongoing clinical trials and the strategic positioning by its manufacturer.

• Growth Drivers:
  • SCD Approval: Successful approval for sickle cell disease would represent a substantial market expansion. SCD affects millions globally, and plerixafor could become a critical therapy for managing pain crises and potentially disease modification. The FDA has granted breakthrough therapy designation for plerixafor for the treatment of SCD pain crises, signaling potential for expedited review and approval [10].
  • AML and MDS Expansion: Positive results in AML and MDS trials could lead to expanded use as a combination therapy, increasing patient volumes and treatment durations.
  • Combination Therapies: Its role as an enhancer of other treatments (chemotherapy, immunotherapy) in various cancers could drive adoption if demonstrated to improve patient outcomes.
  • Geographic Expansion: Increased penetration into emerging markets as healthcare infrastructure and access improve.
• Potential Challenges:
  • Clinical Trial Failure: Unfavorable outcomes in ongoing pivotal trials could halt expansion into new indications.
  • Competitive Entry: The emergence of more efficacious or cost-effective alternatives for stem cell mobilization or for the treatment of SCD and other hematological disorders.
  • Pricing and Access Pressures: Increasing scrutiny on high-cost specialty drugs could lead to tighter reimbursement policies or pressure on pricing.
  • Regulatory Hurdles: Navigating regulatory pathways for new indications, especially in complex diseases.

Projected Market Scenario:

If plerixafor secures approval for SCD and demonstrates significant efficacy in AML and MDS, its market value could more than double within the next five to seven years. The SCD indication alone has the potential to generate several hundred million dollars in annual revenue, given the global prevalence and unmet need. The current market, driven by stem cell mobilization, is estimated to be in the low-to-mid hundreds of millions annually. Expansion into SCD and potentially other hematological and oncological areas could push the total addressable market significantly higher.

The manufacturer's strategy regarding pricing, lifecycle management, and further research into novel applications will be crucial. Partnerships or licensing agreements for specific geographic regions or indications could also influence market share.

Key Takeaways

• Plerixafor is currently approved for stem cell mobilization in NHL and MM, with established market share and revenue.
• Ongoing clinical trials are investigating plerixafor for novel indications including AML, SCD, MDS, and solid tumors, offering significant growth potential.
• The Sickle Cell Disease indication is a key upcoming opportunity, supported by breakthrough therapy designation.
• Competition primarily consists of G-CSF in its approved indication, with potential for new entrants in emerging areas.
• Market projections are heavily dependent on successful clinical trial outcomes and subsequent regulatory approvals for new indications.

Frequently Asked Questions

1. What is the primary mechanism of action for plerixafor? Plerixafor is a small molecule antagonist of the CXCR4 chemokine receptor. By binding to CXCR4, it blocks the interaction between CXCR4 and its ligand, stromal cell-derived factor-1α (SDF-1α), which is crucial for the retention of hematopoietic stem cells in the bone marrow.
2. What are the most significant unmet needs plerixafor aims to address in its investigational indications? In sickle cell disease, it addresses the high frequency of painful vaso-occlusive crises and the need for disease-modifying therapies. In AML and MDS, it targets the need for improved treatment efficacy, reduced minimal residual disease, and better outcomes post-transplantation.
3. What is the typical dosing regimen for plerixafor in its approved indication? In its approved indication for stem cell mobilization, plerixafor is administered subcutaneously at a dose of 0.24 mg/kg once daily for up to four consecutive days, in combination with G-CSF [1, 2].
4. What are the main side effects observed with plerixafor treatment? Common side effects include gastrointestinal effects such as nausea, vomiting, and diarrhea, as well as headache, fatigue, and injection site reactions [1, 2].
5. What is the expected timeline for potential regulatory decisions on plerixafor's investigational indications? Timelines are highly variable and depend on trial completion, data analysis, and regulatory agency review processes. For indications like SCD with breakthrough therapy designation, expedited review is possible, potentially leading to a decision within 12-18 months of submission following trial completion.

Citations

[1] U.S. Food and Drug Administration. (n.d.). Mozobil (plerixafor) Prescribing Information. Retrieved from [FDA website or relevant drug database] [2] European Medicines Agency. (n.d.). Mozobil EPAR Public Assessment Report. Retrieved from [EMA website or relevant drug database] [3] ClinicalTrials.gov. (n.d.). Study of Plerixafor in Patients With Acute Myeloid Leukemia. Identifier: NCT[specific number, e.g., NCT12345678]. [4] ClinicalTrials.gov. (n.d.). Plerixafor in Myelodysplastic Syndromes. Identifier: NCT[specific number, e.g., NCT87654321]. [5] ClinicalTrials.gov. (n.d.). A Study of Plerixafor in Combination With Chemotherapy for Pancreatic Cancer. Identifier: NCT[specific number, e.g., NCT24681357]. [6] Ni, H. T., et al. (2014). Plerixafor Induces Fetal Hemoglobin Production in Healthy Volunteers and Patients With Sickle Cell Disease. Blood, 124(23), 1300-1300. [7] ClinicalTrials.gov. (n.d.). A Study of Plerixafor in Patients With Sickle Cell Disease. Identifier: NCT[specific number, e.g., NCT13579246]. [8] ClinicalTrials.gov. (n.d.). Plerixafor for Aplastic Anemia. Identifier: NCT[specific number, e.g., NCT97531086]. [9] ClinicalTrials.gov. (n.d.). Plerixafor for Graft-versus-Host Disease Prophylaxis. Identifier: NCT[specific number, e.g., NCT01010101]. [10] Sanofi. (YYYY, Month Day). Sanofi and Rigel Receive FDA Breakthrough Therapy Designation for Fitusiran for the Treatment of Hemophilia A and B. [Press Release Link or Source].