NIPENT (pentostatin)
NIPENT is indicated for the treatment of Hairy Cell Leukemia; Anemia; Neutropenia; Thrombocytopenia.
Development Insights
Details
- Status
- Prescription
- First Approved
- 1991-10-11
- Routes
- INJECTION
- Dosage Forms
- INJECTABLE
NIPENT Approval History
What NIPENT Treats
4 indicationsNIPENT is approved for 4 conditions since its original approval in 1991. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Hairy Cell Leukemia
- Anemia
- Neutropenia
- Thrombocytopenia
NIPENT Boxed Warning
WARNING NIPENT should be administered under the supervision of a physician qualified and experienced in the use of cancer chemotherapeutic agents. The use of higher doses than those specified (see DOSAGE AND ADMINISTRATION ) is not recommended. Dose-limiting severe renal, liver, pulmonary, and CNS toxicities occurred in Phase 1 studies that used NIPENT at higher doses (20-50 mg/m 2 in divided doses over 5 days) than recommended. In a clinical investigation in patients with refractory chronic lym...
WARNING NIPENT should be administered under the supervision of a physician qualified and experienced in the use of cancer chemotherapeutic agents. The use of higher doses than those specified (see DOSAGE AND ADMINISTRATION ) is not recommended. Dose-limiting severe renal, liver, pulmonary, and CNS toxicities occurred in Phase 1 studies that used NIPENT at higher doses (20-50 mg/m 2 in divided doses over 5 days) than recommended. In a clinical investigation in patients with refractory chronic lymphocytic leukemia using NIPENT at the recommended dose in combination with fludarabine phosphate, 4 of 6 patients entered in the study had severe or fatal pulmonary toxicity. The use of NIPENT in combination with fludarabine phosphate is not recommended.
NIPENT Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
What's emerging in NIPENT's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications NIPENT treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to NIPENT
3 of 20FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
17 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| 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 |
| NCT03663933 results posted | 180135 18-C-0135 | Ph 2 | active not recruiting | Allogeneic Hematopoietic Cell Transplantation for Disorders of T-cell Proliferation and/or Dysregulation |
| NCT03249831 | 16453 | Ph 1 | active not recruiting | A Blood Stem Cell Transplant for Sickle Cell Disease |
| NCT05757310 | 23139 NCI-2022-10251, 23139 | Ph 1 | recruiting | A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients With Refractory or Recurrent Severe Aplastic Anemia |
| NCT03112603 results posted | INCB 18424-365 (REACH3) CINC424D2301, 2016-004432-38 | Ph 3 | completed | A Study of Ruxolitinib vs Best Available Therapy (BAT) in Patients With Steroid-refractory Chronic Graft vs. Host Disease (GvHD) After Bone Marrow Transplantation (REACH3) |
| NCT01059786 results posted | 100025 10-C-0025 | Ph 2 | active not recruiting | Randomized Phase II Trial of Rituximab With Either Pentostatin or Bendamustine for Multiply Relapsed or Refractory Hairy Cell Leukemia |
| NCT01352312 | 11P.166 2011-23, OFT114197 | Ph 1 | terminated | Combination of Pentostatin, Bendamustine and Ofatumumab for Treatment of Chronic Lymphocytic Leukemia and Lymphoma |
| NCT02105766 results posted | 140077 14-H-0077 | Ph 2 | active not recruiting | Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure |
| NCT00816413 | 0164-07-FB P30CA036727 | Ph 1, Ph 2 | withdrawn | Donor Stem Cell Transplant, Pentostatin, and Total-Body Irradiation in Treating Patients With Hematological Cancer |
| NCT00602836 results posted | CDR0000582676 P30CA015083, MC0784 | Ph 2 | completed | Rituximab, Pentostatin, Cyclophosphamide, and Lenalidomide in Treating Patients With Previously Untreated B-Cell Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma |
| NCT01362790 results posted | 110160 11-C-0160 | Ph 1, Ph 2 | completed | SS1P and Pentostatin Plus Cyclophosphamide for Mesothelioma |
| NCT01024010 results posted | MC0983 NCI-2009-01437, MC0983 | Ph 2 | completed | Ofatumumab, Pentostatin, and Cyclophosphamide in Treating Patients With Untreated Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma |
| NCT00923845 results posted | 080088 08-C-0088 | Ph 2 | completed | Low-Intensity Stem Cell Transplantation With Multiple Lymphocyte Infusions to Treat Advanced Kidney Cancer |
| NCT00816595 results posted | CDR0000630491 P30CA015083, RC0783 | Ph 2 | completed | Pentostatin, Cyclophosphamide, and Rituximab With or Without Bevacizumab in Treating Patients With B-Cell Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma |
| NCT01681563 | PCO 2010-022332-37 | Ph 2 | completed | Trial of Pentostatin Plus Cyclophosphamide With Ofatumumab (PCO) in Older Patients With Chronic Lymphocytic Leukemia |
| NCT00669318 results posted | LS0881 LS0881, 08-000673 | Ph 2 | completed | Pentostatin, Alemtuzumab, and Rituximab in Treating Patients With Relapsed or Refractory Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma |
| NCT01188798 results posted | MUDSIB | Ph 3 | completed | Methotrexate or Pentostatin for Graft-versus-host Disease Prophylaxis in Risk-adapted Allogeneic Bone Marrow Transplantation for Hematologic Malignancies |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
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.
NIPENT FDA Label Details
Indications & Usage
FDA Label (PDF)NIPENT is indicated for the treatment of Hairy Cell Leukemia; Anemia; Neutropenia; Thrombocytopenia.
WARNING NIPENT should be administered under the supervision of a physician qualified and experienced in the use of cancer chemotherapeutic agents. The use of higher doses than those specified (see DOSAGE AND ADMINISTRATION ) is not recommended. Dose-limiting severe renal, liver, pulmonary, and CNS t...
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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