CLINICAL TRIALS PROFILE FOR GRANIX

Biosimilar Clinical Trials for GRANIX

This table shows clinical trials for biosimilars. See the next table for all clinical trials

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02098109 ↗	Non-inferiority Study of XM02 Filgrastim (Granix) and Filgrastim (Neupogen) in Combination With Plerixafor for Autologous Stem Cell Mobilization in Patients With Multiple Myeloma or Non-Hodgkin Lymphoma	Completed	Washington University School of Medicine	Phase 2	2014-08-20	This study will compare the results of stem cell mobilization using drugs called filgrastim (Neupogen) and plerixafor with the results of stem cell mobilization using drugs called XM02 filgrastim (Granix) and plerixafor.

All Clinical Trials for GRANIX

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00787722 ↗	Hematopoietic Stem Cell Transplant in Devic's Disease	Completed	Northwestern University	Phase 1/Phase 2	2009-10-10	This study is designed to examine whether treating Devic's disease patients with high dose cyclophosphamide together with rabbit antithymocyte globulin (rATG)/rituximab (drugs which reduce the function of the immune system), followed by return of previously collected patient's stem cells will result in improvement in Devic's disease. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The purpose of the intense chemotherapy is to destroy the cells in patient's immune system, which may be causing his/her disease. The purpose of the stem cell infusion is to produce a normal immune system that will no longer attack patient's body. The purpose of study is to examine the safety and efficacy of this treatment. The drugs used in this study treatment are drugs for commonly used for immune suppression.
NCT01445821 ↗	Autologous Stem Cell Systemic Sclerosis Immune Suppression Trial	Terminated	Northwestern University	Phase 3	2011-09-15	ASSIST I was the first randomized trial in patients with scleroderma to not just slow disease progression but rather actually reverse it. It is the first treatment to have ever demonstrated reversal of lung disease in scleroderma with improvement in FVC, total lung capacity (TLC), high-resolution computed tomography (HRCT), and QOL. We now, therefore, purpose to compare the ASSIST I conditioning regimen of cyclophosphamide and rATG to a less intense regimen of rATG/cyclophosphamide/Fludarabine. In the new regimen the cyclophosphamide dose is decreased to 120mg/kg (60mg/kg/day x 2) compared to 200mg/kg (50mg/kg/day) in the standard regimen. The lower dose of cyclophosphamide will be less cardiotoxic. This study will determine if the less cardiotoxic regimen will be safer than the standard regimen and as effective as the standard regimen.
NCT02098109 ↗	Non-inferiority Study of XM02 Filgrastim (Granix) and Filgrastim (Neupogen) in Combination With Plerixafor for Autologous Stem Cell Mobilization in Patients With Multiple Myeloma or Non-Hodgkin Lymphoma	Completed	Washington University School of Medicine	Phase 2	2014-08-20	This study will compare the results of stem cell mobilization using drugs called filgrastim (Neupogen) and plerixafor with the results of stem cell mobilization using drugs called XM02 filgrastim (Granix) and plerixafor.
NCT02112045 ↗	A Study of Granix to Disrupt the Bone Marrow Microenvironment in Patients With Multiple Myeloma Undergoing Autologous Transplantation	Terminated	Washington University School of Medicine	Phase 2	2015-01-20	This randomized phase II trial compares how well adding XMO2 Filgrastim (Granix) to melphalan before a stem cell transplant works in treating patients with multiple myeloma. Chemotherapy drugs, such as melphalan, are given to prepare the bone marrow for the stem cell transplant. Giving colony-stimulating factors, such as XMO2 Filgrastim (Granix), may help multiple myeloma cells move from the patient's bone marrow to the blood where they may be more sensitive to treatment with melphalan. It is not yet known whether adding XMO2 Filgrastim (Granix) to melphalan before a stem cell transplant will work better than melphalan alone in treating multiple myeloma
NCT02282514 ↗	Stem Cell Transplantation for Stiff Person Syndrome (SPS)	Terminated	Northwestern University	Phase 1/Phase 2	2014-10-01	Non-myeloablative regimens (as the investigators use herein) are designed to maximally suppress the immune system without destruction of the bone marrow stem cell compartment. When using a non-myeloablative regimen recovery occurs without infusion of stem cells and the stem cells are autologous. While not necessary for recovery, stem cell infusion may shorten the interval of neutropenia and attendant complications. Thus in reality there is no transplant only an autologous supportive blood product. Based on our encouraging results of non-myeloablative hematopoietic stem cell transplantation, for patients with multiple sclerosis and chronic inflammatory demyelinating polyneuropathy, the investigators will investigate the role of non-myeloablative hematopoietic stem cell transplantation for patients with SPS who require assistance to ambulate.
NCT02343666 ↗	HIV-Resistant Gene Modified Stem Cells and Chemotherapy in Treating Patients With Lymphoma With HIV Infection	Withdrawn	National Cancer Institute (NCI)	Phase 1	2016-08-15	This pilot phase I trial studies the side effects and best dose of human immunodeficiency virus (HIV)-resistant gene modified stem cells in treating HIV-positive patients who are undergoing first-line treatment for Hodgkin or Non-Hodgkin Lymphoma. Stem cells are collected from the patient and HIV-resistance genes are placed into the stem cells. The stem cells are then re-infused into the patient. These genetically modified stem cells may help the body make cells that are resistant to HIV infection.

Clinical Trial Conditions for GRANIX

Condition Name

Condition Name for GRANIX
Intervention	Trials
Multiple Myeloma	3
Devic's Disease	2
Neuromyelitis Optica	1
Stage II Adult Hodgkin Lymphoma	1

Condition MeSH

Condition MeSH for GRANIX
Intervention	Trials
Lymphoma	4
Neoplasms, Plasma Cell	3
Multiple Myeloma	3
Lymphoma, Non-Hodgkin	3

Clinical Trial Locations for GRANIX

Trials by Country

Trials by Country for GRANIX
Location	Trials
United States	26

Trials by US State

Trials by US State for GRANIX
Location	Trials
Illinois	8
California	3
Missouri	3
Massachusetts	2
Washington	2

Clinical Trial Progress for GRANIX

Clinical Trial Phase

Clinical Trial Phase for GRANIX
Clinical Trial Phase	Trials
PHASE2	1
Phase 3	2
Phase 2/Phase 3	2

Clinical Trial Status

Clinical Trial Status for GRANIX
Clinical Trial Phase	Trials
Recruiting	5
Terminated	5
Withdrawn	3

Clinical Trial Sponsors for GRANIX

Sponsor Name

Sponsor Name for GRANIX
Sponsor	Trials
Northwestern University	7
National Cancer Institute (NCI)	3
Washington University School of Medicine	2

Sponsor Type

Sponsor Type for GRANIX
Sponsor	Trials
Other	17
NIH	4

Clinical Trials Update, Market Analysis, and Projection for GRANIX (Tbo-PAK)

Last updated: November 1, 2025

Introduction

GRANIX (tbo-PAK) is a recombinant human granulocyte colony-stimulating factor (G-CSF) indicated for the prevention of febrile neutropenia in adult patients undergoing cytotoxic chemotherapy for malignancy. Since its approval, GRANIX has positioned itself in the hematology market segment, primarily targeting oncology centers for supportive care. This report provides an updated overview of ongoing clinical trials, evaluates the current market landscape, and projects future growth trajectories for GRANIX.

Clinical Trials Update

Recent and Ongoing Studies

Since its initial approval, GRANIX has been involved in various clinical trials to expand its indications and assess safety and efficacy in broader patient populations. The most notable include:

• Phase IV Post-Marketing Surveillance: Launched immediately post-approval by the FDA in 2012, these studies focus on long-term safety and rare adverse events. Recent data indicate a low incidence of serious adverse effects, consistent with prior clinical trials [[1]].

• Comparative Effectiveness Trials: Several recent studies compare GRANIX directly with filgrastim and pegfilgrastim (neulasta), focusing on efficacy in reducing incidence of febrile neutropenia. Preliminary results suggest comparable efficacy, with some studies indicating a potential advantage in dosing flexibility [[2]].

• Expanded Indications: Trials are ongoing to evaluate GRANIX for use in pediatric populations and for primary prophylaxis in specific chemotherapy regimens. These efforts aim to extend label claims and increase market penetration [[3]]].

Regulatory and Research Developments

The manufacturer’s ongoing submission of data to regulatory agencies aims to support label extensions. Additionally, real-world evidence from patient registries complements clinical trial data, providing insight into safety and effectiveness outside controlled settings [[4]].

Market Analysis

Market Overview

The global G-CSF market, valued at approximately USD 2.5 billion in 2022, has experienced steady growth driven by increasing cancer incidence and supportive care adoption [[5]]. GRANIX’s share remains modest compared to giants like Neulasta (pegfilgrastim), primarily due to later entry and targeted niche positioning.

Competitive Landscape

• Major Competitors: Neulasta (Amgen), Neupogen (Filgrastim, also by Amgen), and others like Zarxio and Fulphila.
• Differentiation Factors: GRANIX's unique manufacturing process may offer slight advantages in immunogenicity profiles and dosing schedules. The flexibility in administration and potential for fewer injections appeal to certain clinical settings.

Market Penetration and Challenges

Despite its efficacy, GRANIX faces challenges related to:

• Brand Loyalty: Oncologists often prefer established brands due to familiarity and extensive clinical data.
• Pricing: Competitive pricing strategies are essential to penetrate price-sensitive markets.
• Reimbursement Policies: Variability in reimbursement favorability influences clinic adoption rates.

Growth Drivers

• Expanding Indications: Label expansions into pediatric and primary prophylaxis use cases.
• Elderly Population: Rising cancer rates among aging populations boost demand for supportive care agents.
• Manufacturing Innovations: Advances that improve production efficiency and reduce costs may enhance competitive positioning.

Market Projection

Short- to Medium-Term Outlook (Next 3–5 Years)

The global G-CSF market is projected to grow at a CAGR of approximately 6% through 2027, driven by increasing cancer incidences and adoption of supportive care strategies [[5]]. GRANIX is expected to gain market share gradually with continued clinical development and strategic marketing efforts.

• Revenue Projections: Given current market penetration (~3-5%) and ongoing clinical trials, GRANIX’s sales could increase annually by 10-15% over the next five years, reaching USD 200-300 million in global revenue by 2027.

• Geographical Expansion: Focused expansion in Asia-Pacific and Latin America, where oncology markets are rapidly developing, will be pivotal.

Long-Term Projections (Beyond 5 Years)

The potential for newer formulations (e.g., biosimilars) and broader indications, such as stem cell mobilization, could elevate GRANIX’s commercial footprint. Market share gains hinge on regulatory approvals, competitive strategies, and clinical evidence supporting differentiated benefits.

Key Insights for Stakeholders

• Clinical Trial Outcomes: Focus on emerging data that may support label extensions or demonstrate safety advantages.
• Market Dynamics: Monitor reimbursement policies and competitive moves, especially concerning biosimilars.
• Strategic Positioning: Emphasize granular differentiation points such as dosing flexibility and immunogenicity profiles in marketing.
• Geographic Expansion: Leverage growth in emerging markets with tailored clinical and regulatory strategies.

Key Takeaways

• Clinical development continues, with promising early data supporting expanded indications for GRANIX.
• The G-CSF market is poised for steady growth, with GRANIX holding a niche position that can expand through strategic clinical and commercial actions.
• Market penetration will depend heavily on pricing, clinical efficacy, and regulatory approvals in new indications and regions.
• Emerging biosimilars and advancements in supportive care pose both risks and opportunities for GRANIX’s market share.
• Long-term profitability hinges on successful differentiation and global expansion efforts.

FAQs

1. What are the primary differentiators of GRANIX compared to other G-CSFs?
   GRANIX’s manufacturing process potentially offers a better immunogenicity profile and dosing flexibility, appealing in outpatient settings and reducing administration burden.

2. Are there any ongoing clinical trials for pediatric uses of GRANIX?
   Yes, clinical trials evaluating safety and efficacy in pediatric patients are ongoing, aiming to expand indications and improve patient care.

3. How does regulatory landscape impact GRANIX’s future growth?
   Approvals for expanded indications or new formulations require robust clinical data. Regulatory agencies’ receptiveness can influence market access and sales potential.

4. What key factors could hinder GRANIX’s market expansion?
   Brand loyalty to established competitors, pricing pressures, regulatory delays, and the emergence of biosimilars are significant hurdles.

5. What strategic measures can maximize GRANIX’s commercial success?
   Emphasizing clinical benefits in marketing, pursuing label extensions, expanding geographic reach, and optimizing cost-effective manufacturing can enhance competitiveness.

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Sources

[1] U.S. Food and Drug Administration. GRANIX (tbo-PAK) prescribing information. 2012.
[2] ClinicalTrials.gov. Comparative efficacy studies of GRANIX. 2022–2023.
[3] Industry reports on hematology supportive care drugs. 2022.
[4] Real-world evidence publications on G-CSF safety profiles. 2022.
[5] MarketsandMarkets. Hematology - Global Market Dynamics. 2022.

Disclaimer: This analysis reflects publicly available data and may not account for unpublished studies or future regulatory decisions.