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.

Clinical Trial Conditions for ACTIMMUNE

Condition Name

Condition Name for ACTIMMUNE
Intervention	Trials
Friedreich's Ataxia	3
Osteopetrosis	2
Friedreich Ataxia	2
Hepatitis B	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

ACTIMMUNE (tacimogene/Interferon gamma-1b) Clinical Trials Update, Market Analysis, and Projection

Last updated: May 7, 2026

What is ACTIMMUNE and what products are included in “market” coverage?

ACTIMMUNE is an injectable formulation of interferon gamma-1b (Actimune; synthetic interferon gamma) marketed by Eisai for chronic granulomatous disease (CGD) and administered as subcutaneous or intramuscular injections. The clinical and market discussion below covers the interferon gamma-1b brand ecosystem under the ACTIMMUNE trade name.

What is the current clinical trial status for ACTIMMUNE?

No actively recruiting, late-stage (Phase 3) ACTIMMUNE trials were found in the standard public clinical-trial registries at the time of the update. Evidence of ongoing or newly initiated interventional testing is limited and largely does not show as active interventional registration in Phase 3 or Phase 4 for new indications.

Clinical program signal: historical and label-driven use

ACTIMMUNE’s clinical footprint is driven by its established role in CGD, with development history centered on prior randomized evidence that supported regulatory approval. Current usage patterns remain label-based rather than dependent on new pivotal trials.

Operational implication

• R&D pipeline risk: Low likelihood of near-term label expansion in a way that changes the commercial trajectory without a new Phase 3 readout.
• Market access dependency: Demand stays tied to payer recognition of interferon gamma-1b for CGD rather than new indication adoption.

What does the CGD market look like, and where does ACTIMMUNE sit?

CGD is an orphan indication defined by inherited NADPH oxidase pathway defects leading to impaired NADPH oxidase activity. ACTIMMUNE is one of the established pharmacologic options aimed at reducing severe infections by boosting immune function via interferon gamma signaling.

Demand drivers

1. Patient population constraint: CGD is rare, limiting absolute volume growth.
2. Therapy permanence: Treatment patterns in CGD can be long-term depending on disease severity, infection history, and clinician practice.
3. Alternative care pathways: Bone marrow transplant and gene-based approaches can reduce ongoing pharmacotherapy need in selected patients, but uptake is not uniform and does not eliminate chronic management needs.

Competitive landscape (pharmacologic and programmatic)

The competitive set for CGD management includes:

• Other immunomodulators/anti-infective strategies used in CGD supportive care.
• Interventions with curative intent (transplant, emerging gene therapies) that can reduce long-term drug dependence for subsets of patients.

ACTIMMUNE’s market role depends on how these alternatives shift patient pathways and payer authorization decisions.

What is the market outlook for ACTIMMUNE under current conditions?

Given the limited evidence of active late-stage new-trial programs and the label-driven nature of ACTIMMUNE demand, the near-term market outlook follows the standard orphan dynamics: slow growth, modest volatility, and sensitivity to payer policy and wholesale acquisition price changes.

Projection framework used

A projection for ACTIMMUNE is best modeled on:

• Orphan patient pool (steady-state)
• Treatment persistence
• Pricing and rebates (trajectory tied to US reimbursement and tender activity where relevant)
• Competitive substitution and care pathway changes
• Manufacturing continuity and supply stability (matters in injectable biologic markets)

What are the key market variables that change ACTIMMUNE revenue?

1) Pricing and contracting

Revenue shifts in rare disease biologics are typically driven by:

• Wholesale price increases
• Contracted net price changes
• Increased payer scrutiny over injectable immunomodulators

2) Treatment adherence and administration setting

ACTIMMUNE is an injectable therapy. Real-world utilization is influenced by:

• Training and injection burden
• Out-of-pocket cost for caregivers and patients
• Clinical monitoring requirements

3) Substitution effects

Substitution happens through:

• Use of transplant/gene therapy for eligible patients
• Shift in practice patterns for infection prophylaxis and immune modulation

4) Supply and manufacturing

Injectable biologics face:

• Batch release timing and distribution constraints
• Cold-chain logistics and distribution interruptions

Market projection: base, down, up scenarios

Without a verified, current-year revenue and unit dataset for ACTIMMUNE from the same source-of-truth set (company filings or an audited market database), only scenario logic can be provided. The market projection is therefore presented as directionality and drivers rather than numeric revenue figures.

Base case (label-stable, low growth)

• Assumption: CGD treated patients remain on long-term therapy; no major new indication enters.
• Outcome: Revenue remains stable to low-single-digit growth, driven mainly by price versus volume.

Downside case (care-pathway substitution)

• Assumption: Higher adoption of transplant/gene therapies in eligible cohorts and tighter payer controls on interferon gamma immunomodulation.
• Outcome: Volume erosion offsets price; net revenue declines.

Upside case (policy support and improved retention)

• Assumption: Favorable payer coverage expands access; improved clinical retention maintains a higher treated share.
• Outcome: Low growth outperforms base, but growth remains constrained by orphan size.

What should investors and R&D planners watch next?

ACTIMMUNE’s commercial thesis depends less on breakthrough trials and more on continuing label recognition and access.

Near-term watchlist

• Any new ACTIMMUNE trial registration for CGD maintenance or comparative strategy
• Updated clinical guideline language affecting interferon gamma use in CGD
• Payer policy changes on CGD immunomodulators and injection coverage
• Competitive pipeline updates in CGD (especially gene-based and curative pathway adoption rates)
• Manufacturing and supply continuity signals

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Key Takeaways

• ACTIMMUNE is an established interferon gamma-1b brand used in chronic granulomatous disease, with the commercial profile dominated by label-based demand.
• The current public clinical-trial landscape shows limited evidence of active late-stage development that would materially change label breadth in the near term.
• The market outlook is orphan-constraint driven: growth is most sensitive to pricing, payer coverage, treatment persistence, and care-pathway substitution (transplant or gene therapy).
• Projections should be framed as scenarios: base case is label-stable with low growth, with downside from pathway substitution and reimbursement tightening.

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FAQs

1) Why does ACTIMMUNE have limited upside from clinical expansion?

The product’s use case is tightly linked to CGD, and public registries do not show a clear stream of new Phase 3 programs that would add major new indications in the near term.

2) What is the main commercial risk for ACTIMMUNE?

Patient substitution toward curative or semi-curative pathways (transplant and gene therapy where applicable) and payer tightening on immunomodulators for CGD.

3) What is the main growth driver for ACTIMMUNE if there are no new indications?

Net price changes and maintaining treated-patient persistence, since volume growth is inherently constrained by CGD’s rarity.

4) How do injection and administration factors affect ACTIMMUNE use?

Caregiver burden, adherence, and reimbursement for injection services can affect real-world persistence and switching decisions.

5) What signals would indicate ACTIMMUNE is gaining or losing market share?

Coverage expansion and guideline alignment would support gains; evidence of shifted care pathways, formulary exclusions, or increased use of curative options would indicate share loss.

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References

[1] Eisai. ACTIMMUNE (tacimogene/interferon gamma-1b) prescribing information. (Product label).
[2] U.S. National Library of Medicine. ClinicalTrials.gov (search results for ACTIMMUNE/interferon gamma-1b).
[3] PubMed. Clinical studies of interferon gamma-1b in chronic granulomatous disease (historical pivotal evidence).
[4] Professional guidance literature on chronic granulomatous disease management (interferon gamma recommendations in CGD).