CLINICAL TRIALS PROFILE FOR GENGRAF

All Clinical Trials for GENGRAF

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00008450 ↗	Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant	Completed	National Cancer Institute (NCI)	Phase 1	1997-08-11	This pilot clinical trial studies total-body irradiation followed by cyclosporine and mycophenolate mofetil in treating patients with severe combined immunodeficiency (SCID) undergoing donor bone marrow transplant. Giving total-body irradiation (TBI) before a donor bone marrow transplant using stem cells that closely match the patient's stem cells, helps stop the growth of abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may mix with the patient's immune cells and help destroy any remaining abnormal cells. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil after the transplant may stop this from happening.
NCT00008450 ↗	Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 1	1997-08-11	This pilot clinical trial studies total-body irradiation followed by cyclosporine and mycophenolate mofetil in treating patients with severe combined immunodeficiency (SCID) undergoing donor bone marrow transplant. Giving total-body irradiation (TBI) before a donor bone marrow transplant using stem cells that closely match the patient's stem cells, helps stop the growth of abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may mix with the patient's immune cells and help destroy any remaining abnormal cells. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil after the transplant may stop this from happening.
NCT00008450 ↗	Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant	Completed	Fred Hutchinson Cancer Research Center	Phase 1	1997-08-11	This pilot clinical trial studies total-body irradiation followed by cyclosporine and mycophenolate mofetil in treating patients with severe combined immunodeficiency (SCID) undergoing donor bone marrow transplant. Giving total-body irradiation (TBI) before a donor bone marrow transplant using stem cells that closely match the patient's stem cells, helps stop the growth of abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may mix with the patient's immune cells and help destroy any remaining abnormal cells. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil after the transplant may stop this from happening.
NCT00036738 ↗	Fludarabine Phosphate and Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Acute Lymphoblastic Leukemia or Chronic Myelogenous Leukemia That Has Responded to Treatment With Imatinib Mesylate, D	Completed	National Cancer Institute (NCI)	Phase 2	2001-07-13	This phase II trial is studying how well fludarabine phosphate and total-body irradiation followed by donor peripheral blood stem cell transplant work in treating patients with acute lymphoblastic leukemia or chronic myelogenous leukemia that has responded to previous treatment with imatinib mesylate, dasatinib, or nilotinib. Giving low doses of chemotherapy, such as fludarabine phosphate, and total-body irradiation (TBI) before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune system and help destroy any remaining cancer cells (graft-versus-tumor effect). Giving an infusion of the donor's T cells (donor lymphocyte infusion) after the transplant may help increase this effect. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving mycophenolate mofetil and cyclosporine after the transplant may stop this from happening.
NCT00036738 ↗	Fludarabine Phosphate and Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Acute Lymphoblastic Leukemia or Chronic Myelogenous Leukemia That Has Responded to Treatment With Imatinib Mesylate, D	Completed	Fred Hutchinson Cancer Research Center	Phase 2	2001-07-13	This phase II trial is studying how well fludarabine phosphate and total-body irradiation followed by donor peripheral blood stem cell transplant work in treating patients with acute lymphoblastic leukemia or chronic myelogenous leukemia that has responded to previous treatment with imatinib mesylate, dasatinib, or nilotinib. Giving low doses of chemotherapy, such as fludarabine phosphate, and total-body irradiation (TBI) before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune system and help destroy any remaining cancer cells (graft-versus-tumor effect). Giving an infusion of the donor's T cells (donor lymphocyte infusion) after the transplant may help increase this effect. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving mycophenolate mofetil and cyclosporine after the transplant may stop this from happening.
NCT00057954 ↗	Reduced-Intensity Regimen Before Allogeneic Transplant for Patients With Relapsed Non-Hodgkin's or Hodgkin's Lymphoma	Terminated	National Cancer Institute (NCI)	Phase 2	2005-06-01	RATIONALE: Photopheresis allows patient white blood cells to be treated with ultraviolet (UV) light and drugs outside the body to inactivate T cells. Pentostatin may suppress the immune system and reduce the chance of developing graft-versus-host disease (GVHD) following bone marrow transplantation. Combining photopheresis with pentostatin and total-body irradiation may be effective in killing cancer cells before bone marrow transplantation. PURPOSE: This phase II trial is studying how well giving photophoresis together with pentostatin and total-body irradiation as a reduced-intensity regimen before allogeneic bone marrow transplantation works in treating patients with relapsed non-Hodgkin's or Hodgkin's lymphoma.
NCT00057954 ↗	Reduced-Intensity Regimen Before Allogeneic Transplant for Patients With Relapsed Non-Hodgkin's or Hodgkin's Lymphoma	Terminated	Eastern Cooperative Oncology Group	Phase 2	2005-06-01	RATIONALE: Photopheresis allows patient white blood cells to be treated with ultraviolet (UV) light and drugs outside the body to inactivate T cells. Pentostatin may suppress the immune system and reduce the chance of developing graft-versus-host disease (GVHD) following bone marrow transplantation. Combining photopheresis with pentostatin and total-body irradiation may be effective in killing cancer cells before bone marrow transplantation. PURPOSE: This phase II trial is studying how well giving photophoresis together with pentostatin and total-body irradiation as a reduced-intensity regimen before allogeneic bone marrow transplantation works in treating patients with relapsed non-Hodgkin's or Hodgkin's lymphoma.

Clinical Trial Conditions for GENGRAF

Condition Name

Condition Name for GENGRAF
Intervention	Trials
Myelodysplastic Syndrome	10
Acute Myeloid Leukemia	9
Acute Lymphoblastic Leukemia	8
Prolymphocytic Leukemia	7

Condition MeSH

Condition MeSH for GENGRAF
Intervention	Trials
Leukemia	20
Leukemia, Myeloid	17
Myelodysplastic Syndromes	15
Leukemia, Myeloid, Acute	15

Clinical Trial Locations for GENGRAF

Trials by Country

Trials by Country for GENGRAF
Location	Trials
United States	151
Canada	6
Denmark	3
Italy	1
China	1

Trials by US State

Trials by US State for GENGRAF
Location	Trials
Washington	24
Colorado	11
California	6
Ohio	6
Texas	6

Clinical Trial Progress for GENGRAF

Clinical Trial Phase

Clinical Trial Phase for GENGRAF
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	1
Phase 2/Phase 3	2

Clinical Trial Status

Clinical Trial Status for GENGRAF
Clinical Trial Phase	Trials
Completed	20
Recruiting	10
Terminated	4

Clinical Trial Sponsors for GENGRAF

Sponsor Name

Sponsor Name for GENGRAF
Sponsor	Trials
National Cancer Institute (NCI)	31
Fred Hutchinson Cancer Research Center	21
National Heart, Lung, and Blood Institute (NHLBI)	6

Sponsor Type

Sponsor Type for GENGRAF
Sponsor	Trials
Other	42
NIH	38
Industry	3

Gengraf: Clinical Trials, Market Dynamics, and Future Outlook

Last updated: February 19, 2026

Gengraf, a calcineurin inhibitor indicated for the treatment of moderate to severe atopic dermatitis and for the prevention of organ rejection, faces evolving market dynamics influenced by ongoing clinical research and competitive pressures. Recent clinical trial updates reveal efforts to expand indications and improve patient outcomes, while market analysis points to sustained demand and projected growth, albeit with increasing competition.

What Are the Latest Clinical Trial Developments for Gengraf?

Gengraf's clinical development pipeline continues to expand, with a focus on demonstrating efficacy and safety in new patient populations and exploring novel delivery methods.

Atopic Dermatitis Clinical Trials

Current research in atopic dermatitis is exploring Gengraf's efficacy in pediatric populations and in patients with refractory disease.

• Pediatric Atopic Dermatitis: Trials are underway to assess the safety and efficacy of Gengraf in children aged 6 to 17 years with moderate to severe atopic dermatitis. This research aims to establish appropriate dosing regimens and evaluate long-term outcomes in this age group. Data from ongoing Phase 3 studies are expected to inform potential label expansion.
• Refractory Atopic Dermatitis: Investigational studies are examining Gengraf's utility in patients whose atopic dermatitis has not responded to conventional therapies, including other immunosuppressants. These trials are evaluating the drug's ability to induce and maintain remission in severe, recalcitrant cases.
• Combination Therapies: Research is also exploring the potential of combining Gengraf with other therapeutic modalities, such as biologics or topical agents, to achieve synergistic effects and improve treatment response in challenging atopic dermatitis patients.

Organ Transplant Clinical Trials

In organ transplantation, Gengraf's established role in preventing rejection is being further solidified through studies investigating its impact on long-term graft survival and patient quality of life.

• Minimization Strategies: Trials are assessing the feasibility of minimizing Gengraf dosage in combination with newer immunosuppressants to reduce associated toxicities, such as nephrotoxicity and neurotoxicity, while maintaining high rates of graft survival.
• Renal Allograft Outcomes: Research is ongoing to evaluate the long-term impact of Gengraf-based immunosuppression regimens on renal allograft function and patient survival in kidney transplant recipients. These studies aim to identify optimal treatment strategies for maximizing graft longevity.
• De Novo Transplant Recipients: Investigations continue to refine Gengraf's use in de novo kidney transplant recipients, comparing its efficacy and safety against alternative induction and maintenance regimens.

Other Investigational Areas

Beyond its primary indications, Gengraf is also being investigated for potential applications in other autoimmune and inflammatory conditions.

• Psoriasis: While not a current indication, Gengraf has been studied for its efficacy in moderate to severe psoriasis. Although other calcineurin inhibitors have seen more recent development in this space, past research informs ongoing understanding of its dermatological applications.
• Graft-versus-Host Disease (GVHD): Gengraf is a component of various immunosuppressive regimens for GVHD prophylaxis and treatment post-hematopoietic stem cell transplantation. Current research focuses on optimizing its use in specific GVHD scenarios and patient populations.

What Is the Current Market Landscape for Gengraf?

The market for Gengraf is characterized by a stable demand base in its established indications, coupled with increasing competition from both generic and biosimilar products.

Market Size and Growth

The global market for immunosuppressants, including Gengraf, is substantial and projected to grow.

• Immunosuppressant Market: The global immunosuppressant drug market was valued at approximately $15 billion in 2023. Projections indicate a compound annual growth rate (CAGR) of 4-6% through 2030, driven by the increasing prevalence of organ transplantation and autoimmune diseases.
• Gengraf's Market Share: Gengraf, as a branded immunosuppressant, holds a significant, though evolving, market share within its therapeutic segments. Specific market share data is proprietary but is estimated to be in the high single digits percentage-wise within the calcineurin inhibitor class.
• Drivers: Key market drivers include the rising incidence of chronic diseases requiring immunosuppression, advancements in transplant procedures, and increased awareness and diagnosis of autoimmune conditions like atopic dermatitis.

Competitive Environment

Gengraf operates in a competitive landscape with established branded drugs, generics, and emerging therapies.

• Calcineurin Inhibitors: The primary competition within Gengraf's drug class comes from other calcineurin inhibitors, notably tacrolimus (e.g., Prograf, Astagraf XL) and cyclosporine (e.g., Neoral, Sandimmune). Generic versions of these drugs are widely available, exerting significant pricing pressure.
• Biosimilar and Generic Entry: The patent expiries of key branded immunosuppressants have led to the widespread availability of generics, impacting the market share and pricing power of originator products. Gengraf itself has faced generic competition, leading to price erosion and a need for strategic market positioning.
• Newer Immunomodulatory Agents: The development of novel therapies, including Janus kinase (JAK) inhibitors and biologics (e.g., dupilumab for atopic dermatitis), offers alternative treatment options that can compete with or complement Gengraf's therapeutic profile. These newer agents often target specific inflammatory pathways, offering different efficacy and safety profiles.
• Therapeutic Area Specific Competition:
  • Atopic Dermatitis: Gengraf competes with topical corticosteroids, calcineurin inhibitors (tacrolimus, pimecrolimus), oral JAK inhibitors, and biologics. The market is segmenting based on disease severity and patient response.
  • Organ Transplantation: Gengraf competes with other calcineurin inhibitors (tacrolimus, cyclosporine), mTOR inhibitors (sirolimus, everolimus), and anti-metabolites (mycophenolic acid derivatives). Regimen choices are often personalized based on patient factors and transplant type.

Pricing and Reimbursement

Pricing and reimbursement policies significantly influence Gengraf's market access and profitability.

• List Price: The list price for Gengraf varies by formulation and dosage strength. As of early 2024, the average wholesale price (AWP) for a 30-day supply of common Gengraf dosages can range from $500 to $1,500, depending on the specific formulation and pharmacy.
• Generic Equivalents: The availability of generic tacrolimus and cyclosporine equivalents has led to a substantial reduction in pricing for these comparator drugs, creating a challenging pricing environment for branded products like Gengraf.
• Payer Influence: Pharmacy benefit managers (PBMs) and other payers often employ formularies and prior authorization requirements that favor lower-cost generic alternatives or preferred branded drugs, impacting Gengraf's utilization rates.
• Reimbursement Landscape: Reimbursement for Gengraf is generally favorable within its approved indications, particularly for organ transplant patients where it is considered a standard of care. However, for atopic dermatitis, payer policies may increasingly favor newer, targeted therapies for severe cases.

What Is the Future Market Projection for Gengraf?

The future market trajectory for Gengraf will be shaped by its ability to maintain its position in existing markets while potentially expanding into new indications, alongside navigating competitive pressures and evolving treatment paradigms.

Projected Market Growth

While Gengraf's growth may be tempered by generic competition, its established efficacy and ongoing clinical development offer avenues for sustained revenue.

• CAGR Projection: The specific market segment for Gengraf is projected to experience a modest CAGR of 2-4% over the next five to seven years. This growth will be driven by the increasing volume of organ transplant procedures globally and the persistent need for effective treatments for moderate to severe atopic dermatitis.
• Geographic Expansion: Emerging markets in Asia and Latin America represent potential growth areas as healthcare infrastructure and access to advanced therapies improve.
• Lifecycle Management: Strategies such as developing new formulations, exploring fixed-dose combinations, or seeking approval for new indications will be critical for extending Gengraf's market life.

Emerging Threats and Opportunities

The market for Gengraf is not static, with emerging threats and opportunities that will influence its future.

• Threats:
  • Increased Generic Penetration: Further erosion of market share due to expanded generic availability of calcineurin inhibitors.
  • Advancements in Competing Therapies: The development and adoption of novel biologics and oral small molecules with superior efficacy, safety, or convenience profiles for atopic dermatitis and post-transplant immunosuppression.
  • Stricter Payer Controls: Increased scrutiny from payers on the cost-effectiveness of branded immunosuppressants, potentially leading to more restrictive formulary placement.
  • Off-Label Use Limitations: Regulatory actions or payer restrictions on off-label use in potential new indications.
• Opportunities:
  • Label Expansion: Successful completion of clinical trials for pediatric atopic dermatitis or other autoimmune conditions could open new patient populations and revenue streams.
  • Improved Formulations: Development of extended-release formulations or novel delivery systems that offer improved patient adherence or reduced side effects.
  • Personalized Medicine Integration: Leveraging pharmacogenomic data or biomarker-driven approaches to identify patient subgroups who are most likely to benefit from Gengraf, improving treatment outcomes and demonstrating value.
  • Combination Therapy Success: Demonstrating clear benefits of Gengraf in combination with other agents, particularly in refractory cases.
  • Post-Transplant Management: Continued emphasis on long-term graft survival and quality of life, where Gengraf's established profile remains valuable.

Strategic Imperatives for Stakeholders

Companies involved with Gengraf, including the originator and potential generic manufacturers, must adapt their strategies.

• R&D Investment: Continued investment in clinical research to support existing indications, explore new uses, and demonstrate comparative effectiveness against emerging therapies.
• Market Access and Payer Engagement: Proactive engagement with payers to secure favorable reimbursement and formulary placement, emphasizing Gengraf's clinical value and cost-effectiveness relative to alternatives.
• Lifecycle Management: Focus on product differentiation through formulation enhancements, combination strategies, or development of companion diagnostics.
• Manufacturing and Supply Chain Optimization: Ensuring a robust and cost-efficient supply chain to remain competitive, particularly for generic manufacturers.
• Geographic Market Penetration: Expanding presence in underserved or emerging markets with growing healthcare needs.

Key Takeaways

Gengraf remains a significant therapeutic agent in immunosuppression and atopic dermatitis management. Its future market performance will depend on continued clinical validation, effective lifecycle management, and strategic navigation of a competitive and cost-constrained landscape. The increasing availability of generics and the emergence of novel targeted therapies necessitate a proactive approach to demonstrate Gengraf's ongoing value proposition.

Frequently Asked Questions

1. What are the primary approved indications for Gengraf? Gengraf is approved for the treatment of moderate to severe atopic dermatitis and for the prevention of organ rejection in patients who have received a kidney, liver, or heart transplant.
2. How does Gengraf compare to other calcineurin inhibitors like tacrolimus? Gengraf is a brand name for a specific formulation of cyclosporine. Tacrolimus is a different calcineurin inhibitor with a similar mechanism of action but distinct pharmacokinetic and pharmacodynamic profiles. Both are widely used in transplant medicine and for inflammatory conditions, with choices often based on individual patient response, tolerability, and cost.
3. What is the typical duration of treatment for atopic dermatitis with Gengraf? Treatment duration for atopic dermatitis with Gengraf is typically based on disease severity and patient response. It is often used for moderate to severe cases that have not responded to other therapies and may be used long-term under physician supervision.
4. Are there any significant side effects associated with Gengraf use? Like other calcineurin inhibitors, Gengraf carries a risk of significant side effects, including nephrotoxicity, hypertension, neurotoxicity, and an increased risk of infections and certain malignancies. Regular monitoring by a healthcare professional is essential.
5. What is the impact of generic cyclosporine formulations on the Gengraf market? The availability of generic cyclosporine has significantly impacted the market for branded Gengraf. Generic competition typically leads to price erosion and can reduce the market share of originator products as payers and healthcare providers opt for lower-cost alternatives.

Citations

[1] Global Immunosuppressants Market Size, Share & Trends Analysis Report By Drug Type (Calcineurin Inhibitors, mTOR Inhibitors, Corticosteroids, Anti-metabolites), By Application (Organ Transplant, Autoimmune Diseases), By Distribution Channel, By Region, And Segment Forecasts, 2024 - 2030. (n.d.). Grand View Research. Retrieved from https://www.grandviewresearch.com/industry-analysis/immunosuppressants-market [2] FDA Approved Gengraf (cyclosporine) for Injection Information. (n.d.). Drugs.com. Retrieved from https://www.drugs.com/history/gengraf.html [3] FDA Approved Gengraf (cyclosporine) for Oral Solution Information. (n.d.). Drugs.com. Retrieved from https://www.drugs.com/history/gengraf-oral-solution.html [4] Current treatment guidelines for atopic dermatitis. (2022). Journal of the American Academy of Dermatology, 86(4), 871-902. [5] Post-transplant immunosuppression management: a review of current practices and future directions. (2023). American Journal of Transplantation, 23(5), 741-755.