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Data updated: May 26, 2026

HYFTOR (sirolimus)

mTOR Inhibitors Genetic Support Trial Activity: Stable 76 active trials
Rare Disease Approved 2022-03-22

HYFTOR is indicated for the treatment of Facial Angiofibroma; Tuberous Sclerosis.

Source: FDA Label • NOBELPHARMA • mTOR Inhibitor Immunosuppressant
Jump to: Indications Approvals Trials Competitors Safety Patents

How HYFTOR Works

The exact mechanism of sirolimus in the treatment of facial angiofibroma is unknown. However, tuberous sclerosis is characterized by genetic defects in TSC1 and TSC2, which lead to the constitutive activation of the mammalian target of rapamycin (mTOR). Sirolimus acts by inhibiting mTOR activation.

Source: FDA Label

Development Insights

National Cancer Institute (NCI) conducting 12 trials (5%)
583 indications explored (Broad Platform)
→ sickle cell disease (14 trials)
→ myelodysplastic syndrome (9 trials)
→ graft versus host disease (8 trials)
1
Indication
--
Phase 3 Trials
1
Priority Reviews
4
Years on Market

Details

Status
Prescription
First Approved
2022-03-22
Patent Cliff
2029

Pro Metrics

Patent cliff and revenue data

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Routes
TOPICAL
Dosage Forms
GEL

Companies

NOBELPHARMA
Active Ingredient: SIROLIMUS

HYFTOR Approval History

2023
2024
2025
2026
Original
New Indication
New Form
Label Update
1 FDA actions from 2022 to 2022
Mar 2022 ORIGINAL Priority
New Form · Type 3 - New Dosage Form
FDA Letter Label

What HYFTOR Treats

2 indications

HYFTOR is approved for 2 conditions since its original approval in 2022. These indications span multiple therapeutic areas including oncology, immunology, and more.

  • Facial Angiofibroma
  • Tuberous Sclerosis
Source: FDA Label

HYFTOR Target & Pathway

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Target

MTOR (Mechanistic Target of Rapamycin) Intracellular Kinase

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Clinical Trial Registry

227 trials
Trial Sponsor ID Phase Status Title
NCT05896839 NCI-2023-04306 NCI-2023-04306, ETCTN10614 Ph 1, Ph 2 recruiting Immunotherapy in Combination With Prednisone and Sirolimus for Kidney Transplant Recipients With Unresectable or Metastatic Skin Cancer
NCT02042326 MAV-RAPA PHRCN10-PR-DEVAUCHELLE 2011-000321-69 Ph 2 recruiting Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous Malformations
NCT06876142 10001811 001811-C Ph 1, Ph 2 suspended Combination Therapy (Mirdametinib and Sirolimus) for RAS Mutated Relapsed Refractory Multiple Myeloma
NCT02629120 160032 16-I-0032 Ph 1, Ph 2 active not recruiting High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease
NCT07581704 202601163 Ph 1 not yet recruiting Sirolimus Pre-conditioning on T Cell Activity and T-cell Engaging Bispecific Antibody Efficacy in Multiple Myeloma
NCT05088356 IRB-60439 NCI-2021-12228 Ph 1 active not recruiting Reduced Intensity Allogeneic HCT in Advanced Hematologic Malignancies w/T-Cell Depleted Graft
NCT07582172 25663 NCI-2026-03208, 25663 Ph 2 not yet recruiting Total Marrow and Lymphoid Irradiation in Combination With Fludarabine and Melphalan as Conditioning for Allogeneic Peripheral Blood Stem Cell Hematopoietic Cell Transplant in Older Patients With Refractory and Relapsed Acute Myeloid Leukemia and High-risk Myelodysplastic Syndrome
NCT05463133 10000977 000977-I Ph 1, Ph 2 recruiting Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists
NCT04888741 MoTD RG_19-116 Ph 2 recruiting Methods of T Cell Depletion Trial (MoTD)
NCT07113743 10000186 000186-I Ph 1, Ph 2 enrolling by invitation Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease
NCT03214354 Sickle-AID TRU-17-001 Ph 2 recruiting Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor
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
NCT06145282 results posted 10001635 001635-H Ph 1, Ph 2 active not recruiting Non-myeloablative Haploidentical HCT Study for Patients With Sickle Cell Disease, Including Compromised Organ Function
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
NCT05436418 10000613 000613-C Ph 1, Ph 2 recruiting The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantation
NCT00977691 results posted 090225 09-H-0225 Ph 1, Ph 2 active not recruiting Haploidentical PBMC Transplant for Severe Congenital Anemias
NCT03077542 results posted 170069 17-H-0069 Ph 1, Ph 2 active not recruiting Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease
NCT07542314 ENHANCE SRP-9001-402 Ph 4 not yet recruiting Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting
NCT06261060 2023-0918 NCI-2024-01333 Ph 2 recruiting Low-Dose Sirolimus to Increase Hematopoietic Function in Patients With RUNX1 Familial Platelet Disorder
NCT04177004 19214 NCI-2019-06454, 19214 Ph 1 suspended Human Lysozyme Goat Milk for the Prevention of Graft Versus Host Disease in Patients With Blood Cancer Undergoing a Donor Stem Cell Transplant
NCT07052929 2957-CL-0101 2025-523213-29 Ph 1, Ph 2 recruiting Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators
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
NCT00811915 EPARGNE 2007/125/HP Ph 3 terminated Study to Compare the Safety and Efficacy of Sirolimus (Rapamune) to Tacrolimus (Advagraf) Associated to Mycophenolate Mofetil (CellCept) Between 12 and 36 Months After Kidney Transplantation
NCT00792948 results posted NCI-2009-00800 NCI-2009-00800, S0805 Ph 2 active not recruiting Combination Chemotherapy With or Without Donor Stem Cell Transplant in Treating Patients With Acute Lymphoblastic Leukemia
NCT06084780 TRANSCAPE CASE7Z23 Ph 2 not yet recruiting Intestinal & Multivisceral Transplantation for Unresectable Mucinous Carcinoma Peritonei (TRANSCAPE)
NCT06325709 10001580 001580-I Ph 1, Ph 2 recruiting Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease
NCT02891603 results posted MCC-18783 5R01HL133823-02 Ph 1, Ph 2 completed A Phase I/II GVHD Prevention Trial Combining Pacritinib With Sirolimus-Based Immune Suppression
NCT03605927 MCC-19305 Ph 1 completed CD40-L Blockade for Prevention of Acute Graft-Versus-Host Disease
NCT04411654 J3Z-MC-OJAB (PRV-GD2-101) Ph 1, Ph 2 active not recruiting Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
NCT06843811 24-022433 Ph 2 enrolling by invitation Sirolimus for Leigh Syndrome
NCT04339101 results posted 19576 NCI-2020-01722, 19576 Ph 2 completed Itacitinib, Tacrolimus, and Sirolimus for the Prevention of GVHD in Patients With Acute Leukemia, Myelodysplastic Syndrome, or Myelofibrosis Undergoing Reduced Intensity Conditioning Donor Stem Cell Transplantation
NCT06959771 10002385 002385-I Ph 1, Ph 2 recruiting Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study
NCT07270549 GAIN-CTNNB1 0120-536/2025-2711 2025-522719-40-00 Ph 1, Ph 2 recruiting Gene Replacement Therapy for Treatment of Paediatric Patients With CTNNB1 Neurodevelopmental Syndrome
NCT04141020 20190857 1R01NS111119-01A1 Ph 2 recruiting Effect of Sirolimus on Molecular Alterations in Cerebral Aneurysms
NCT04127578 J3Z-MC-OJAA formerly PRV-PD101 Ph 1, Ph 2 active not recruiting Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)
NCT05357482 10000539 000539-H Ph 1, Ph 2 active not recruiting Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia
NCT03970096 RG1005364 9749, NCI-2019-03188 Ph 2 recruiting Graft Versus Host Disease-Reduction Strategies for Donor Blood Stem Cell Transplant Patients With Acute Leukemia or Myelodysplastic Syndrome (MDS)
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
NCT05386914 2091042 Ph 1 active not recruiting This Study Consists of Two Study Parts Conducted Under a Single IRB. Part I: Short Term ApoE-dependent Cerebral Blood Flow Response to Sirolimus in Cognitively Normal Adults Part II: Short Term ApoE-dependent Cerebral Blood Flow and Lung Perfusion Response to Sirolimus in Cognitively Normal Adults
NCT04861064 00106369 Ph 2 recruiting Weekly Sirolimus Therapy
NCT03933904 results posted 832465 Ph 2 active not recruiting Sirolimus in Previously Treated Idiopathic Multicentric Castleman Disease
NCT06055608 ADVANTage DAIT CTOT-41 Ph 2 recruiting Advancing Transplantation Outcomes in Children
NCT02574728 AflacST1502 IRB00082488 Ph 2 active not recruiting Sirolimus in Combination With Metronomic Chemotherapy in Children With Recurrent and/or Refractory Solid and CNS Tumors
NCT04469530 AflacST1903 STUDY00000113 Ph 2 recruiting Sirolimus in Combination With Metronomic Chemotherapy in Children With High-Risk Solid Tumors
NCT05170828 PRESERVE Ph 1 withdrawn Cryopreserved MMUD BM With PTCy for Hematologic Malignancies
NCT07426484 25-743 Ph 4 not yet recruiting Cosibelimab for CSCC in Patients With Kidney Transplant or Hematologic Malignancy
NCT01203722 results posted J1055 NA_00039823, P01CA015396 Ph 1, Ph 2 completed Reduced Intensity, Partially HLA Mismatched BMT to Treat Hematologic Malignancies

Showing 50 of 227 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

ClinicalTrials.gov ↗ Pro

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.

HYFTOR FDA Label Details

Indications & Usage

FDA Label (PDF)

HYFTOR is indicated for the treatment of Facial Angiofibroma; Tuberous Sclerosis.

HYFTOR Patents & Exclusivity

Exclusivity: Mar 2029

Exclusivity

ODE-391 Until Mar 2029
Source: FDA Orange Book

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Data Sources

FDA Approval: NDA213478 → Label: DailyMed → Patents: FDA Orange Book → Trials: ClinicalTrials.gov →

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