CLINICAL TRIALS PROFILE FOR MOZOBIL

All Clinical Trials for Mozobil

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.
NCT00103662 ↗	Mobilization of Stem Cells With AMD3100 (Plerixafor) in Multiple Myeloma 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, generic name of filgrastim) is better than G-CSF alone to mobilize and collect the optimal number of stem cells in multiple myeloma patients for autologous transplantation.
NCT00322387 ↗	Mobilization of Stem Cells With Plerixafor, Chemotherapy and G-CSF in Multiple Myeloma or Non-Hodgkin's Lymphoma Patients	Completed	Genzyme, a Sanofi Company	Phase 2	2004-04-01	Patients with multiple myeloma (MM) and non-Hodgkin's lymphoma (NHL) will be mobilized with chemotherapy and G-CSF plus plerixafor (AMD3100). The purpose of this protocol is to determine if plerixafor given after chemotherapy and G-CSF mobilization regimen is safe, if it can increase the circulating levels of peripheral blood stem cells (PBSCs) by ≥ 2-fold before apheresis, and if transplantation with the apheresis product was successful, as measured by time to engraftment of polymorphonuclear leukocytes (PMNs) and platelets (PLTs).

Clinical Trial Conditions for Mozobil

Condition Name

Condition Name for Mozobil
Intervention	Trials
Multiple Myeloma	18
Lymphoma	5
Lymphoma, Non-Hodgkin	5
Acute Myeloid Leukemia	5

Condition MeSH

Condition MeSH for Mozobil
Intervention	Trials
Multiple Myeloma	22
Neoplasms, Plasma Cell	20
Lymphoma, Non-Hodgkin	20
Lymphoma	19

Clinical Trial Locations for Mozobil

Trials by Country

Trials by Country for Mozobil
Location	Trials
United States	151
Canada	10
Italy	5
Germany	3
France	2

Trials by US State

Trials by US State for Mozobil
Location	Trials
California	17
Missouri	12
New York	11
Washington	11
Massachusetts	8

Clinical Trial Progress for Mozobil

Clinical Trial Phase

Clinical Trial Phase for Mozobil
Clinical Trial Phase	Trials
PHASE2	1
PHASE1	1
Phase 4	2

Clinical Trial Status

Clinical Trial Status for Mozobil
Clinical Trial Phase	Trials
Completed	53
Terminated	13
Recruiting	10

Clinical Trial Sponsors for Mozobil

Sponsor Name

Sponsor Name for Mozobil
Sponsor	Trials
Genzyme, a Sanofi Company	30
National Cancer Institute (NCI)	13
Washington University School of Medicine	6

Sponsor Type

Sponsor Type for Mozobil
Sponsor	Trials
Other	96
Industry	45
NIH	25

Last updated: May 23, 2026

Mozobil (plerixafor): clinical trials update, market analysis and projection (2026–2035)

Executive summary: Mozobil (plerixafor) is a mobilization drug used in combination with G-CSF for hematopoietic stem cell mobilization. Public disclosures show a late-lifecycle product with a mature clinical evidence base and limited new interventional trial activity relative to newer competitors in hematopoietic stem cell mobilization. Commercial trajectory is driven by (1) transplant volumes, (2) physician uptake of on-label single-use mobilization workflows, and (3) competitive substitution risk from alternative CXCR4 antagonists and G-CSF-only strategies. With current market structure, Mozobil’s revenue outlook is constrained by market maturity and patent/brand aging, but downside is moderated by entrenched clinician experience and the narrow indication.

Note: This response contains only information that can be cited from publicly available sources within the provided knowledge boundary. Where a specific trial endpoint, sponsor, or market forecast figure is not directly supported by a citable source, it is omitted.

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What is Mozobil (plerixafor) and what is it approved for?

Mozobil is the brand name for plerixafor (CXCR4 antagonist). It is indicated, with G-CSF, to mobilize hematopoietic progenitor cells for collection and subsequent autologous transplantation in patients with non-Hodgkin’s lymphoma and multiple myeloma.

Key approved use (U.S.)

• FDA labeling: use with G-CSF for mobilization of CD34+ hematopoietic progenitor cells prior to autologous transplant in specified indications (as described in the prescribing information). (FDA label; see references)

Dosing and regimen

• Mozobil is administered as a dosing regimen in the mobilization window after G-CSF priming, followed by leukapheresis for stem cell collection (FDA label). (FDA label; see references)

What clinical endpoints support approval?

The pivotal clinical development program used transplant-relevant mobilization endpoints:

• CD34+ cell mobilization in peripheral blood
• Successful collection (target CD34+ cells)
• Engraftment-related outcomes post-transplant

These are summarized in FDA review materials and the prescribing information. (FDA label; see references)

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What clinical trials update exists for Mozobil (plerixafor)?

Status: mature evidence base, limited visible late-stage interventional expansion Most Mozobil clinical evidence is historical and anchored to approval-grade studies. Public FDA labeling materials and older registration cohorts document efficacy through mobilization and collection success, with subsequent safety characterization across the use population. (FDA label; see references)

What is the most recent public “trial-like” activity footprint?

The public footprint for Mozobil after launch largely appears through:

• Postmarketing safety reporting and pharmacovigilance summaries
• Registry-based clinical use publications, rather than new phase 3 pivotal trials
• Comparative mobilization discussions in guideline and review settings

No specific phase 3 “registration-changing” Mozobil trial update can be cited from the available sources in this response.

What trial designs would be expected for future Mozobil updates (but without claiming they exist)?

A typical future clinical program in mobilization would focus on:

• Comparative mobilization efficacy vs other CXCR4 antagonists or other mobilization algorithms
• Patient subgroup performance (older adults, poor mobilizers, comorbidity strata)
• Real-world collection success and time-to-transplant benchmarks

This section is descriptive only and does not assert active trials.

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How big is the hematopoietic stem cell mobilization market and where does Mozobil fit?

Market segmentation logic for projection

Mozobil sits inside the broader hematopoietic stem cell mobilization market, which is shaped by:

1. Autologous transplant volumes in multiple myeloma and non-Hodgkin’s lymphoma
2. Physician preference for mobilization protocols and time-to-collection outcomes
3. Use of chemotherapy-free vs chemotherapy-based mobilization
4. Institutional protocols for “poor mobilizer” rescue strategies

Why transplant volumes drive Mozobil demand

Demand for mobilization drugs scales with:

• The number of patients undergoing apheresis for autologous transplantation
• Adoption of mobilization workflows that include CXCR4 antagonists

Those volumes can be approximated using published transplant epidemiology and cancer treatment statistics; however, a fully cited numeric market size model cannot be produced in this response without market-size sources that are not included in the provided data.

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What companies compete with Mozobil in stem cell mobilization?

Direct drug-class competition

The closest substitution risk is other CXCR4 antagonists used in mobilization settings, alongside algorithmic substitutions using G-CSF-based approaches.

In the absence of fully citable, current competitive launch and market share data in this response, the competitive set is best handled by category:

• CXCR4 antagonists used for mobilization
• G-CSF-only mobilization protocols
• Chemomobilization workflows that reduce need for CXCR4 antagonists

How does competition typically affect Mozobil pricing and volume?

Competitive pressure tends to shift:

• Market share in centers adopting alternative CXCR4 antagonists
• Relative usage in “poor mobilizer” strategies
• Formulary and payer utilization

No specific MoZobil market share decline figures are cited here.

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What is the Orange Book status of Mozobil (plerixafor)?

Mozobil is a branded product. Orange Book exclusivity and patent listings determine generic entry risk. Without an Orange Book record included in the provided data set for this response, the exact patent-by-patent status cannot be listed reliably.

What matters for generic entry risk

For small-molecule brands like plerixafor, generic entry hinges on:

• Patent expiration for active ingredient and composition-of-matter coverage
• Method-of-use and formulation patents
• Regulatory exclusivities that can delay ANDA approvals

This response does not assert specific listed patents or expiration dates.

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When does Mozobil lose exclusivity and what generic entry risks exist?

A generic entry risk model requires:

• Confirmed Orange Book listings (patent numbers and expiration dates)
• Whether patents remain listed at the time of analysis
• Any relevant litigation or settlement history

No such patent table is included here because it would require Orange Book and litigation source data that are not present in the provided dataset.

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What patent litigation affects Mozobil (plerixafor)?

A litigation-impact analysis requires citations to court dockets, filings, or settlement terms tied to ANDA/Paragraph IV events. No citable litigation dataset is available in the provided inputs for this response, so this section is not populated.

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What formulations are protected for Mozobil and does delivery format drive IP barriers?

For plerixafor, the main commercial form is an injectable solution used for mobilization dosing. IP protection commonly includes:

• Composition-of-matter around the drug substance
• Formulation and stability related to the injectable matrix
• Manufacturing process patents

This response does not list specific formulation or process patents because a citable patent register is not included.

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How does Mozobil compare with other mobilization agents on clinical outcomes?

Mozobil is positioned as a CXCR4 antagonist that enhances mobilization efficacy when added to G-CSF. In the pivotal evidence base described in labeling:

• It increases CD34+ cell yields and collection success compared with historical mobilization outcomes without CXCR4 blockade
• Safety is characterized through adverse event profiles documented in the prescribing information

Detailed head-to-head comparative trial results versus each competitor drug are not itemized here because citable comparative trial datasets are not provided.

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Market projection for Mozobil (plerixafor) through 2035: base, bull, bear drivers

Projection framework (what will move the number)

Revenue drivers for a transplant-support drug like Mozobil:

• Transplant incidence in myeloma and NHL (autologous segment)
• Apheresis capacity and protocol intensity (centers with established mobilization workflows)
• Adoption rate of CXCR4 antagonist addition to G-CSF
• Therapy substitution toward other agents if clinically equivalent options exist
• Reimbursement and contracting in major transplant networks
• Generic or biosimilar-like competitive replacement if patents and exclusivity erode

Numerical projection

A numeric 2026–2035 revenue forecast requires market-size starting points, transplant epidemiology time series, and pricing/trajectory assumptions tied to cited data. Those cited inputs are not present in the provided data set. As a result, this response does not produce numerical projections.

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Key takeaways

• Mozobil (plerixafor) is a G-CSF combination mobilization drug for autologous hematopoietic stem cell transplantation in specified myeloma and NHL settings, supported by mobilization and collection outcomes in labeling. (FDA label; see references)
• The drug’s clinical evidence base is mature, with public “trial update” activity appearing more consistently in post-approval use and safety reporting than in new pivotal phase 3 expansion, based on the available cited material in this response. (FDA label; see references)
• Market growth is primarily a function of autologous transplant volumes and adoption of CXCR4 antagonist-containing mobilization protocols.
• A defensible numeric market projection for 2026–2035 requires Orange Book patent status, competitive market share, and transplant incidence time series with citations, which are not included in the provided dataset, so numeric forecasts are not stated.

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FAQs

1. Is Mozobil (plerixafor) used for mobilizing stem cells in both lymphoma and multiple myeloma?
   Mozobil is indicated with G-CSF for hematopoietic progenitor cell mobilization prior to autologous transplantation in specified non-Hodgkin’s lymphoma and multiple myeloma populations per FDA labeling. (FDA label)

2. What is Mozobil’s role versus G-CSF alone in stem cell mobilization?
   Mozobil is used with G-CSF to improve mobilization outcomes, particularly CD34+ cell yield and successful collection, as reflected in labeled clinical evidence. (FDA label)

3. Does Mozobil require prior chemotherapy for mobilization?
   FDA labeling specifies use in combination with G-CSF for mobilization in the labeled settings. Whether chemotherapy is part of a patient’s mobilization regimen depends on the institution and the labeled population context described in the prescribing information. (FDA label)

4. What determines generic entry risk for Mozobil?
   Generic entry risk is determined by Orange Book-listed patents and any applicable exclusivities, plus Paragraph IV challenge outcomes and litigation. Specific listings are not enumerated in this response.

5. Where does Mozobil face the most substitution risk commercially?
   Substitution risk is greatest where centers use alternative mobilization algorithms or other CXCR4 antagonists and where payer contracting limits branded utilization, but a quantified competitive assessment is not provided in this response.

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References (APA)

1. U.S. Food and Drug Administration. (n.d.). Mozobil (plerixafor) prescribing information. FDA. https://www.accessdata.fda.gov/ (FDA label)