TheraRadar
Data updated: May 26, 2026

CELLCEPT (mycophenolate mofetil)

Genetic Support Trial Activity: Mature 2 active trials
Immunology Approved 1995-05-03

CellCept (mycophenolate mofetil) is an antimetabolite immunosuppressant indicated for the prophylaxis of organ rejection in adult and pediatric patients aged 3 months and older receiving allogeneic kidney, heart, or liver transplants. It is intended for use in a multi-drug regimen, typically in combination with corticosteroids and a calcineurin inhibitor.

Source: FDA Label • Roche

How CELLCEPT Works

Mycophenolate mofetil (MMF) is a prodrug that is hydrolyzed to its active metabolite, mycophenolic acid (MPA). MPA is a selective, uncompetitive, and reversible inhibitor of inosine monophosphate dehydrogenase (IMPDH), which inhibits the *de novo* pathway of guanosine nucleotide synthesis. Because T- and B-lymphocytes are critically dependent on *de novo* synthesis for proliferation—whereas other cell types can utilize salvage pathways—MPA exerts potent cytostatic effects on lymphocytes. This results in suppressed proliferative responses, reduced antibody production, and inhibited recruitment of immune cells to the transplanted organ.

Development Insights

Dartmouth-Hitchcock Medical Center conducting 1 trials (14%)
16 indications explored (Broad Platform)
acute myeloid leukemia (2 trials)
kidney transplantation (2 trials)
chronic lymphocytic leukemia (1 trials)
8
Indications
--
Phase 3 Trials
2
Priority Reviews
31
Years on Market

Details

Status
Prescription
First Approved
1995-05-03
Revenue
$110M (Q4-2025)

Pro Metrics

Patent cliff and revenue data

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Routes
ORAL, INJECTION
Dosage Forms
TABLET, FOR SUSPENSION, INJECTABLE, CAPSULE

Companies

Active Ingredient: MYCOPHENOLATE MOFETIL

CELLCEPT Approval History

1996
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2012
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2016
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2026
Original
New Indication
New Form
Label Update
167 FDA actions from 1995 to 2025 · 3 indication expansions
Jun 2025 SUPPL
Label · Labeling
Aug 2024 SUPPL
Update · REMS
Aug 2022 SUPPL
Label · Labeling

What CELLCEPT Treats

1 indications

CELLCEPT is approved for 1 conditions since its original approval in 1995. These indications span multiple therapeutic areas including oncology, immunology, and more.

  • Organ Rejection
Source: FDA Label

CELLCEPT Boxed Warning

EMBRYOFETAL TOXICITY, MALIGNANCIES and SERIOUS INFECTIONS Use during pregnancy is associated with increased risks of first trimester pregnancy loss and congenital malformations. Avoid if safer treatment options are available. Females of reproductive potential must be counseled regarding pregnancy prevention and planning [see Warnings and Precautions (5.1) , Use in Special Populations (8.1 , 8.3) ] . Increased risk of development of lymphoma and other malignancies, particularly of the skin [see W...

CELLCEPT Target & Pathway

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Target

MTOR (Mechanistic Target of Rapamycin) Intracellular Kinase

CELLCEPT Competitive Set

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Three rings of competition based on shared molecular targets and treated indications.

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Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.

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Clinical Trial Registry

265 trials
Trial Sponsor ID Phase Status Title
NCT07524530 10002233 002233-C Ph 2 not yet recruiting Stem Cell Transplantation for Participants With Germline RUNX1 Associated Blood Cancers
NCT06996119 23822 NCI-2025-03603, 23822 Ph 1 recruiting Emapalumab With Post-Transplant Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil for the Prevention of Graft-versus-Host Disease After Donor Reduced-Intensity Hematopoietic Cell Transplant
NCT07566377 26-168 Ph 2 recruiting Cord Blood Transplantation in Children and Young Adults With Blood Cancer
NCT04888741 MoTD RG_19-116 Ph 2 recruiting Methods of T Cell Depletion Trial (MoTD)
NCT05039619 POSTERITY WA42985 2021-000097-29, 2023-505825-15-00 Ph 2 recruiting A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Obinutuzumab in Adolescents With Active Class III or IV Lupus Nephritis and the Safety and PK of Obinutuzumab in Pediatric Participants
NCT03670966 RG1003349 10060, P30CA015704 Ph 1, Ph 2 recruiting 211At-BC8-B10 Followed by Donor Stem Cell Transplant in Treating Patients With Relapsed or Refractory High-Risk Acute Leukemia or Myelodysplastic Syndrome
NCT01231412 results posted 2448.00 NCI-2010-02035, 2448.00 Ph 3 completed Graft-Versus-Host Disease Prophylaxis in Treating Patients With Hematologic Malignancies Undergoing Unrelated Donor Peripheral Blood Stem Cell Transplant
NCT05327023 10000489 000489-C Ph 1, Ph 2 recruiting Donor Lymphocyte Infusion After Allogeneic Hematopoietic Cell Transplantation for High-Risk Hematologic Malignancies
NCT07162038 10001785 001785-C Ph 1 recruiting Phase I Trial Integrating HLA-Haploidentical Anti-CD19 CAR-T Cells With Post-Transplantation Cyclophosphamide-Based HLA-Haploidentical Hematopoietic Cell Transplantation
NCT04959175 10000359 000359-C Ph 1, Ph 2 recruiting Phase I/II Study to Reduce Post-transplantation Cyclophosphamide Dosing for Older or Unfit Patients Undergoing Bone Marrow Transplantation for Hematologic Malignancies
NCT06807606 2024-1605 NCI-2025-00794 Ph 2 recruiting Optimized Cord Blood Transplantation for the Treatment of Patients With High-risk Hematologic Malignancies Who Have Relapsed After First Allogeneic Stem Cell Transplantation
NCT07412470 FREXERA EFC18554 2025-521521-33-00 Ph 2, Ph 3 recruiting A Study to Investigate the Efficacy and Safety of Frexalimab Versus Tacrolimus in Adults Undergoing Kidney Transplantation
NCT07548983 STUDY00028015 NCI-2025-09157, STUDY00028015 Ph 1 not yet recruiting Ruxolitinib With Azacitidine Maintenance for the Treatment of Patients With Acute Myeloid Leukemia Undergoing Reduced Intensity Allogeneic Stem Cell Transplantation
NCT06973668 2024-1940 NCI-2025-03239 Ph 2 recruiting A Randomized Study to Compare Post-transplant Cyclophosphamide, Sirolimus, Ruxolitinib and Post-transplant Cyclophosphamide, Sirolimus, Mycophenolate Mofetil to Prevent Graft Versus Host Disease
NCT07493538 2025LS162 Ph 2 recruiting MT2025-35 Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning Treosulfan and Fludarabine, With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases
NCT07349771 MC230810 24-009803 Ph 2 not yet recruiting Axatilimab Plus Standard of Care Therapy for the Prevention of Graft Versus Host Disease Following Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Hematologic Cancer, ABRAXAS Trial
NCT01384513 results posted 11D.247 2011-31, JT 1795 Ph 2 completed A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies
NCT04384692 RG1006507 NCI-2020-01626, 10093 Ph 2 active not recruiting Peritransplant Ruxolitinib for Patients With Primary and Secondary Myelofibrosis
NCT03192397 results posted I 44417 NCI-2017-01069, I 44417 Ph 1, Ph 2 active not recruiting Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant
NCT04547049 NFD-001 Ph 3 active not recruiting A Study Comparing Haploidentical Hematopoietic Stem Cell Transplantations (HSCTs) From Young Non-first-degree and Older First-degree Donors in Hematological Malignancies
NCT05736419 23-009 Ph 2 recruiting A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
NCT02333162 IRB14-0709 NCI-2014-02469, IRB14-0709 Ph 1 suspended Intensity Modulated Total Marrow Irradiation, Fludarabine Phosphate, and Melphalan in Treating Patients With Relapsed Hematologic Cancers Undergoing a Second Donor Stem Cell Transplant
NCT02251821 results posted 9033 NCI-2014-01882, 9033 Ph 2 completed JAK Inhibitor Before Donor Stem Cell Transplant in Treating Patients With Primary or Secondary Myelofibrosis
NCT01659606 12-950 IRB-P00003466 Ph 2 active not recruiting Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita
NCT05538208 PLUMM ML42264 1R01AR079124-01A1 Ph 2 recruiting The Pediatric Lupus Nephritis Mycophenolate Mofetil (PLUMM) Study
NCT04904588 ACCESS ACCESS Ph 2 active not recruiting HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide
NCT04644016 20-480 Ph 2 recruiting Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders
NCT05622318 PRO00046962 Ph 2 active not recruiting De-escalated Cyclophosphamide (PTCy) and Ruxolitinib for Graft-versus-Host Disease (GVHD) Prophylaxis
NCT06752694 RG1124040 NCI-2024-06524, 20575 Ph 2 recruiting Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult Patients With Acquired Aplastic Anemia
NCT06872333 2024LS140 Ph 2 recruiting Allo HSCT for High Risk Hemoglobinopathies
NCT04375631 RG1006914 NCI-2020-02616, RG1006914 Ph 1 recruiting CLAG-M or FLAG-Ida Chemotherapy and Reduced-Intensity Conditioning Donor Stem Cell Transplant for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelomonocytic Leukemia
NCT03249831 16453 Ph 1 active not recruiting A Blood Stem Cell Transplant for Sickle Cell Disease
NCT06055608 ADVANTage DAIT CTOT-41 Ph 2 recruiting Advancing Transplantation Outcomes in Children
NCT04195633 RG1005742 NCI-2019-07697, 10343 Ph 2 recruiting Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Total-Body Irradiation for the Treatment of Hematological Malignancies
NCT05170828 PRESERVE Ph 1 withdrawn Cryopreserved MMUD BM With PTCy for Hematologic Malignancies
NCT03480360 results posted D17170 NCI-2018-01157 Ph 3 active not recruiting Haploidentical Allogeneic Peripheral Blood Transplantation: Examining Checkpoint Immune Regulators' Expression
NCT01203722 results posted J1055 NA_00039823, P01CA015396 Ph 1, Ph 2 completed Reduced Intensity, Partially HLA Mismatched BMT to Treat Hematologic Malignancies
NCT07359859 25-212 Ph 2 recruiting A Study of Ruxolitinib for Preventing Graft-Versus-Host Disease in People With a Hematologic Malignancy Who Will Receive a Stem Cell Transplant
NCT05193565 B110_02KT2103 Ph 4 active not recruiting Study to Compare the Efficacy and Safety After Conversion to RaparoBell® or My-Rept® in Kidney Transplant Patients
NCT02861417 2016-0137 NCI-2017-00614, 2016-0137 Ph 2 active not recruiting Busulfan, Fludarabine Phosphate, and Post-Transplant Cyclophosphamide in Treating Patients With Blood Cancer Undergoing Donor Stem Cell Transplant
NCT03504241 TEACH DAIT ITN062ST UM1AI109565, NIAID CRMS ID#: 20676 Ph 1 completed Tolerance by Engaging Antigen During Cellular Homeostasis
NCT06001385 OPTIMIZE OPTIMIZE Ph 2 active not recruiting HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation With Reduced Dose Post Transplantation Cyclophosphamide GvHD Prophylaxis
NCT04877288 IM103-402 2022-501677-39 Ph 3 recruiting A Study to Evaluate the Benefits and Risks of Conversion of Existing Adolescent Kidney Transplant Recipients Aged 12 to <18 Years to a Belatacept-based Immunosuppressive Regimen as Compared to Continuation of a Calcineurin Inhibitor-based Regimen, and Their Adherence to Immunosuppressive Medications
NCT00882895 09-0042 / 201101864 Ph 2 active not recruiting Tandem Stem Cell Transplantation for Non-Hodgkin's Lymphoma
NCT03032783 16D.606 JT 9551 Ph 2 completed A Two Step Approach to Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic Malignancies-Increasing GVT Effects Without Increasing Toxicity
NCT04262843 19518 NCI-2019-08984, 19518 Ph 2 active not recruiting Total Marrow and Lymphoid Irradiation as Conditioning Regimen Before Hematopoietic Cell Transplantation in Patients With Myelodysplastic Syndrome or Acute Leukemia
NCT02081755 013-307 Ph 4 enrolling by invitation Safety and Efficacy of Everolimus Treatment in Liver Transplantation for Liver Cancer
NCT03121001 2016-1152 Ph 2 recruiting Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease
NCT06013423 RG1123652 NCI-2023-05598, FHIRB0020219 Ph 2 recruiting Cord Blood Transplant, Cyclophosphamide, Fludarabine, and Total-Body Irradiation in Treating Patients With High-Risk Hematologic Diseases
NCT03292861 ATG Pilot Study Ph 2 enrolling by invitation The Effect and Safety Profile of Thymoglobulin® in Primary Cardiac Transplant Recipients

Showing 50 of 265 trials

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Active Pipeline

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Key Completed Trials

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Completed studies with published results, ranked by significance

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Trial Timeline

Full development history with FDA approval milestones

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Understanding FDA Approval Types
Count Type What it means
- ORIG Original approval - drug first enters market
- SUPPL - Efficacy New indication (new disease/condition approved)
- SUPPL - Labeling Label text changes (warnings, dosing updates)
- SUPPL - Manufacturing Production changes (new facility)
- SUPPL - Chemistry Formulation changes (new dosage strength)

Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.

CELLCEPT FDA Label Details

Indications & Usage

FDA Label (PDF)

CELLCEPT is indicated for the treatment of Organ Rejection.

⚠️ BOXED WARNING

WARNING: EMBRYOFETAL TOXICITY, MALIGNANCIES and SERIOUS INFECTIONS Use during pregnancy is associated with increased risks of first trimester pregnancy loss and congenital malformations. Avoid if safer treatment options are available. Females of reproductive potential must be counseled regarding pre...

Pro Intelligence Preview

Deep insights for CELLCEPT

Revenue Insights

  • Q4-2025: $110M
  • Historical trend analysis

Patent Timeline

  • Patent expiration dates
  • Generic/biosimilar risk

Trial Analysis

  • 7 total trials
  • Stage: Mature

Competitive Landscape

  • 16 similar drugs
  • Same target/indication analysis
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Data Sources

Data sourced from official FDA and NIH databases. Click links to verify on original sources.

How We Calculate These Metrics

Trial Activity Stage

Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.

Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.

  • Growth: High proportion of early-phase trials (Phase 1/2), active development
  • Expansion: Significant Phase 3 activity, approaching or pursuing approvals
  • Mature: Substantial Phase 4 post-marketing studies
  • Stable: Mixed phase distribution, steady development
  • Declining: Low active trial ratio, reduced R&D investment