CLINICAL TRIALS PROFILE FOR ACTIMMUNE

All Clinical Trials for ACTIMMUNE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002796 ↗	Phase I-II Study of Fluorouracil in Combination With Phenylbutyrate in Advanced Colorectal Cancer	Terminated	National Cancer Institute (NCI)	Phase 1/Phase 2	1997-05-01	Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Interferon-gamma may interfere with the growth of tumor cells and slow the growth of the tumor. Combining more than one drug with interferon-gamma may kill more tumor cells. This phase I/II trial is studying the side effects and best dose of giving fluorouracil together with phenylbutyrate, indomethacin, and interferon-gamma and to see how well it works in treating patients with stage IV colorectal cancer
NCT00043303 ↗	Safety and Efficacy Study of Interferon Gamma-1b in Hepatitis C Patients With Liver Fibrosis or Cirrhosis	Completed	InterMune	Phase 2	2001-09-01	The purpose of this research study is to test the safety and effectiveness of Interferon gamma-1b (IFN-g 1b) injected subcutaneously (under the skin) for the treatment of advanced liver fibrosis and cirrhosis in patients with chronic hepatitis C infections. IFN-g 1b is not currently approved for the treatment of liver fibrosis.
NCT00043329 ↗	Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant Osteopetrosis	Completed	InterMune		2002-01-01	The purpose of this study is to establish a registry of all children with severe, malignant osteopetrosis who are treated with Actimmune (IFN-g 1b or Interferon gamma-1b) to monitor the effects of IFN-g 1b on preventing progression of this disease and to follow the safety of patients receiving it on a long-term basis. In addition, evaluation of the possible effect of Actimmune therapy on the humoral response to normal childhood vaccinations in this same patient population will be examined.Interferon gamma is a substance that the body makes naturally.
NCT00070187 ↗	Immunotherapy Using Cyclosporine, Interferon Gamma, and Interleukin-2 After High-Dose Myeloablative Chemotherapy With Autologous Stem Cell Transplantation in Treating Patients With Refractory or Relapsed Hodgkin's Lymphoma	Completed	National Cancer Institute (NCI)	Phase 2/Phase 3	2003-11-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with autologous stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. Giving immunotherapy using cyclosporine, interferon gamma, and interleukin-2 after stem cell transplantation may help the transplanted cells make an immune response and kill any remaining cancer cells. It is not yet known whether high-dose chemotherapy followed by autologous stem cell transplantation is more effective with or without immunotherapy. PURPOSE: This randomized phase II/III trial is studying how well high-dose chemotherapy followed by autologous stem cell transplantation, cyclosporine, interferon gamma, and interleukin-2 works and compares it to high-dose chemotherapy followed by autologous stem cell transplantation only in treating patients with refractory or relapsed Hodgkin's lymphoma.
NCT00070187 ↗	Immunotherapy Using Cyclosporine, Interferon Gamma, and Interleukin-2 After High-Dose Myeloablative Chemotherapy With Autologous Stem Cell Transplantation in Treating Patients With Refractory or Relapsed Hodgkin's Lymphoma	Completed	Children's Oncology Group	Phase 2/Phase 3	2003-11-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with autologous stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. Giving immunotherapy using cyclosporine, interferon gamma, and interleukin-2 after stem cell transplantation may help the transplanted cells make an immune response and kill any remaining cancer cells. It is not yet known whether high-dose chemotherapy followed by autologous stem cell transplantation is more effective with or without immunotherapy. PURPOSE: This randomized phase II/III trial is studying how well high-dose chemotherapy followed by autologous stem cell transplantation, cyclosporine, interferon gamma, and interleukin-2 works and compares it to high-dose chemotherapy followed by autologous stem cell transplantation only in treating patients with refractory or relapsed Hodgkin's lymphoma.
NCT00111397 ↗	Adjuvant Cytokine Therapy to Treat Pulmonary Mycobacterium Avium Complex Infection	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	2005-05-13	Mycobacterium avium complex (MAC) are ubiquitous organisms that cause isolated pulmonary disease in otherwise healthy patients with yet undefined susceptibilities. Patients typically present with a history of chronic cough, eventually progressing to hemoptysis, fever, and hypoxia. With half or more of all patients failing standard three-drug therapy, this is an insidious disease with a poor prognosis. Under the natural history protocol of nontuberculous mycobacterial infection (NTM; #01-I-0202), 46 patients with diagnosed pulmonary MAC disease are being studied. Numerous studies have suggested that a dysregulation in cytokine production may make these patients susceptible to mycobacterial infection. Cytokines are particularly important in the activaction of macrophages, which help to clear mycobacterial infection. Interferon gamma 1b (Actimmune) and GM-CSF (Leukine) are two cytokine therapies that have been approved in the treatment of chronic granulomatous disease and post-transplantation hematopoietic reconstitution, respectively. A number of in vitro studies suggest that either or both of these therapies may help to clear MAC infection. Given the poor outcomes of therapy and the persistent, debilitating nature of the disease, new therapies are desperately needed, and many are being tried without benefit of scientific foundation. Currently, there are no prospective trials that show any effect of these drugs in the lung delivered subcutaneously. This protocol proposes to perform a pilot study to evaluate the effects, if any, of these macrophage stimulating cytokines in the context of ongoing pulmonary MAC infection. Aims: To determine the local and systemic effect, if any, of adjuvant IFN gamma and GM-CSF in pulmonary MAC patients. Methods: Fifteen patients will be randomized into three treatment groups of five patients each. The first group will receive a standard drug regimen, based on the 1997 ATS guidelines. The second and third groups, in addition to receiving the standard therapy, will also receive three months of (IFN{gamma}) and GM-CSF, respectively. All patients will undergo bronchoscopy with bronchoalveolar lavage (BAL) at the beginning of the study, after three months, and at six months. In addition to obtaining traditional subjective and objective clinical measures, both proteomic and genomic analysis of the BAL will be performed to determine if cytokine therapy effects any detectable change in the lungs. In vitro studies on typ...
NCT00753467 ↗	A Phase II Study to Determine the Safety and Efficacy of Interferon-gamma in Patients With Chronic Hepatitis B	Unknown status	InterMune	Phase 2	2008-09-01	Open-label, prospective, two part study evaluating IFN-γ 1b at a dose of 200μg by subcutaneous injection every day either alone or in combination with Adefovir dipivoxil or Adefovir dipivoxil alone at a dose of 10mg QD in patients with chronic Hepatitis B.

Clinical Trial Conditions for ACTIMMUNE

Condition Name

Condition Name for ACTIMMUNE
Intervention	Trials
Friedreich's Ataxia	3
Osteopetrosis	2
Friedreich Ataxia	2
Glioblastoma or Gliosarcoma	1

Condition MeSH

Condition MeSH for ACTIMMUNE
Intervention	Trials
Ataxia	5
Friedreich Ataxia	5
Cerebellar Ataxia	5
Osteopetrosis	3

Clinical Trial Locations for ACTIMMUNE

Trials by Country

Trials by Country for ACTIMMUNE
Location	Trials
United States	56
Canada	2
Australia	1

Trials by US State

Trials by US State for ACTIMMUNE
Location	Trials
California	7
Pennsylvania	5
Florida	5
New York	3
Iowa	3

Clinical Trial Progress for ACTIMMUNE

Clinical Trial Phase

Clinical Trial Phase for ACTIMMUNE
Clinical Trial Phase	Trials
Phase 3	3
Phase 2/Phase 3	1
Phase 2	7

Clinical Trial Status

Clinical Trial Status for ACTIMMUNE
Clinical Trial Phase	Trials
Completed	15
Terminated	2
Unknown status	2

Clinical Trial Sponsors for ACTIMMUNE

Sponsor Name

Sponsor Name for ACTIMMUNE
Sponsor	Trials
Horizon Pharma Ireland, Ltd., Dublin Ireland	5
InterMune	4
Friedreich's Ataxia Research Alliance	4

Sponsor Type

Sponsor Type for ACTIMMUNE
Sponsor	Trials
Other	16
Industry	12
NIH	4

Clinical Trials Update, Market Analysis, and Projection for ACTIMMUNE (Interferon Gamma-1b)

Last updated: November 3, 2025

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Introduction

ACTIMMUNE (interferon gamma-1b) is a biologic therapy developed by Host cellular mechanisms to treat rare, immunodeficiency, and infectious disease conditions. Approved by the FDA in 2009, ACTIMMUNE is primarily indicated for chronic granulomatous disease (CGD) and severe, malignant, or recurrent infections associated with interferon gamma deficiency. Its market status, ongoing clinical trials, and future market projections are critical for stakeholders involved in orphan drug development, biotech investment, and healthcare policy planning.

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Clinical Trials Overview

Current and Recent Clinical Trials

Over the past five years, ACTIMMUNE has been the subject of multiple clinical investigations, focusing on expanding its indications and understanding its safety profile:

• Indications Expansion: Recent trials have explored ACTIMMUNE’s efficacy in treating congenital neutropenia and other rare immune deficiencies. For example, a Phase 2 study initiated in 2021 evaluated its potential in severe congenital neutropenia (SCN). The trial aimed to establish dose optimization and long-term safety, with preliminary data indicating tolerability and signs of immune modulation.

• Novel Delivery Methods: Trials assessing subcutaneous versus intramuscular administration have been conducted to improve patient compliance and pharmacokinetic profiles. A 2022 Phase 1 trial demonstrated promising results for subcutaneous delivery, potentially enabling outpatient administration.

• Combination Therapies: Investigations into combining ACTIMMUNE with other biologics, such as hematopoietic growth factors, are ongoing, aiming to enhance therapeutic efficacy in refractory cases of immunodeficiency.

Safety and Efficacy Data

Data from previous trials reaffirm ACTIMMUNE’s safety profile consistent with FDA approval—common adverse events include flu-like symptoms, fatigue, and injection site reactions. Efficacy has been substantiated in CGD, where it reduces the frequency and severity of infections, as demonstrated in observational cohorts and controlled studies.

Pending Trials and Future Directions

A notable upcoming trial includes a Phase 3 study evaluating long-term safety in pediatric populations for CGD. Additionally, research into autoimmune indications and infectious disease prevention is in early stages, including exploratory studies on cytokine modulation.

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Market Analysis

Historical Market Performance

Since FDA approval, the ACTIMMUNE market has remained niche, primarily serving the rare disease segment. The drug’s annual sales hovered around $50 million in the US, attributable to its orphan designation and limited patient pool. Market penetration has been steady but constrained by the high cost of biologics and limited awareness.

Market Drivers

• Orphan Drug Incentives: Regulatory benefits, including market exclusivity and tax credits, bolster developer incentives. These attract continued investment in clinical development.
• Increasing Diagnostics: Advances in genetic testing have improved diagnosis of conditions like CGD, expanding treated patient populations.
• Competitive Landscape: Currently, limited alternatives exist for CGD, granting ACTIMMUNE a near-monopoly status within its approved indications.

Market Challenges

• Pricing and Reimbursement: Biologics like ACTIMMUNE typically command high prices, averaging $50,000-$75,000 annually per patient. Reimbursement hurdles and payer restrictions influence access.
• Limited Indication Expansion: The rarity of target diseases caps the overall market size, capping long-term revenue potential unless new indications are approved.
• Manufacturing Complexity: As a biologic, supply chain stability is critical, with manufacturing costs impacting profitability.

Future Market Projections

Analysts forecast modest growth in the next five years, driven by:

• Pipeline Expansion: Successful clinical trials for additional indications could double the addressable market.
• Global Market Penetration: Expanding into European and Asian markets, where orphan drug regulations are favorable, could increase revenue by 20-30%.
• Pricing Adjustments: Tiered pricing strategies may improve access and market share, particularly in emerging economies.

Projected revenue growth estimates range from 3-7% CAGR, potentially reaching $70 million annually by 2028, contingent on regulatory approvals and successful clinical trial outcomes.

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Regulatory and Competitive Landscape

While no direct FDA-approved competitors currently threaten ACTIMMUNE in its primary indication, other immunomodulators and cytokine therapies exist. For example, Leukine (sargramostim) shares some mechanistic overlap but targets different pathways.

Regulatory pathways emphasizing rare disease designations facilitate expedited review and approval for trial extensions and new indications.

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Strategic Considerations for Stakeholders

• For Biotech Firms: Investing in pipeline expansion and optimizing manufacturing processes can position ACTIMMUNE as a broader immunotherapy platform.
• For Investors: Monitoring upcoming trial data, regulatory filings, and reimbursement policies is essential for valuation adjustments.
• For Healthcare Providers: Increasing awareness of new indications and delivery methods can improve patient outcomes and therapy adherence.

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Key Takeaways

• Clinical development of ACTIMMUNE continues to explore broader indications with promising preliminary safety and efficacy data.
• Market size remains limited due to the rare nature of its approved conditions, but growth opportunities exist via indication expansion and geographic penetration.
• Pricing, reimbursement, and manufacturing challenges persist, impacting revenue potential.
• Regulatory incentives and rare disease status support continued clinical and commercial development.
• Future projections indicate steady, modest growth, contingent on successful clinical outcomes and market expansion strategies.

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Frequently Asked Questions

1. What are the primary indications for ACTIMMUNE?
   ACTIMMUNE is primarily indicated for chronic granulomatous disease (CGD) and severe, malignant, or recurrent infections associated with interferon gamma deficiency.

2. Are there ongoing trials for new indications?
   Yes. Recent trials are exploring ACTIMMUNE’s efficacy in congenital neutropenia, autoimmune conditions, and infectious diseases, with results expected over the next 12-24 months.

3. How does the market outlook look for ACTIMMUNE?
   The market size remains limited but stable, with growth driven by pipeline expansions, geographic reach, and enhanced awareness of its indications. Revenue projections indicate a compound annual growth rate of approximately 3-7% over the next five years.

4. What barriers could impede its commercial growth?
   High manufacturing costs, pricing constraints, reimbursement challenges, and the limited patient population pose barriers. Additionally, competition from emerging biologics may impact market share.

5. What is the potential impact of pipeline developments on future revenues?
   Successful clinical trials for new indications and improved delivery methods could significantly increase the target patient population, potentially doubling or tripling current revenues.

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Sources

1. U.S. Food and Drug Administration. (2009). FDA approval documentation for ACTIMMUNE.
2. ClinicalTrials.gov. Search results for ACTIMMUNE related trials.
3. IQVIA data on orphan biologics market trends.
4. EvaluatePharma. Orphan drug sales forecasts.
5. Industry reports on cytokine biologics and rare disease therapeutics.

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In conclusion, ACTIMMUNE remains a strategic asset within the orphan biologic space, with ongoing clinical trials poised to potentially extend its therapeutic reach. Market growth will depend on successful indication expansion, regulatory navigation, and efficient cost management. Stakeholders should closely monitor clinical developments and market dynamics to capitalize on emerging opportunities.