CLINICAL TRIALS PROFILE FOR NIPENT

505(b)(2) Clinical Trials for NIPENT

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Combination	NCT03249831 ↗	A Blood Stem Cell Transplant for Sickle Cell Disease	Recruiting	California Institute for Regenerative Medicine (CIRM)	Phase 1	2019-01-04	Blood stem cells can produce red blood cells (which carry oxygen), white blood cells of the immune system (which fight infections) and platelets (which help the blood clot). Patients with sickle cell disease produce abnormal red blood cells. A blood stem cell transplant from a donor is a treatment option for patients with severe sickle cell disease. The donor can be healthy or have the sickle cell trait. The blood stem cell transplant will be given to the patient as an intravenous infusion (IV). The donor blood stem cells will then make normal red blood cells - as well as other types of blood cells - in the patient. When blood cells from two people co-exist in the patient, this is called mixed chimerism. Most children are successfully treated with blood stem cells from a sibling (brother/sister) who completely shares their tissue type (full-matched donor). However, transplant is not an option for patients who (1) have serious medical problems, and/or (2) do not have a full-matched donor. Most patients will have a relative who shares half of their tissue type (e.g. parent, child, and brother/sister) and can be a donor (half-matched or haploidentical donor). Adult patients with severe sickle cell disease were successfully treated with a half-matched transplant in a clinical study. Researchers would like to make half-matched transplant an option for more patients by (1) improving transplant success and (2) reducing transplanted-related complications. This research transplant is being tested in this Pilot study for the first time. It is different from a standard transplant because: 1. Half-matched related donors will be used, and 2. A new combination of drugs (chemotherapy) that does not completely wipe out the bone marrow cells (non-myeloablative treatment) will be used to prepare the patient for transplant, and 3. Most of the donor CD4+ T cells (a type of immune cells) will be removed (depleted) before giving the blood stem cell transplant to the patient to improve transplant outcomes. It is hoped that the research transplant: 1. Will reverse sickle cell disease and improve patient quality of life, 2. Will reduce side effects and help the patient recover faster from the transplant, 3. Help the patient keep the transplant longer and 4. Reduce serious transplant-related complications.
New Combination	NCT03249831 ↗	A Blood Stem Cell Transplant for Sickle Cell Disease	Recruiting	City of Hope Medical Center	Phase 1	2019-01-04	Blood stem cells can produce red blood cells (which carry oxygen), white blood cells of the immune system (which fight infections) and platelets (which help the blood clot). Patients with sickle cell disease produce abnormal red blood cells. A blood stem cell transplant from a donor is a treatment option for patients with severe sickle cell disease. The donor can be healthy or have the sickle cell trait. The blood stem cell transplant will be given to the patient as an intravenous infusion (IV). The donor blood stem cells will then make normal red blood cells - as well as other types of blood cells - in the patient. When blood cells from two people co-exist in the patient, this is called mixed chimerism. Most children are successfully treated with blood stem cells from a sibling (brother/sister) who completely shares their tissue type (full-matched donor). However, transplant is not an option for patients who (1) have serious medical problems, and/or (2) do not have a full-matched donor. Most patients will have a relative who shares half of their tissue type (e.g. parent, child, and brother/sister) and can be a donor (half-matched or haploidentical donor). Adult patients with severe sickle cell disease were successfully treated with a half-matched transplant in a clinical study. Researchers would like to make half-matched transplant an option for more patients by (1) improving transplant success and (2) reducing transplanted-related complications. This research transplant is being tested in this Pilot study for the first time. It is different from a standard transplant because: 1. Half-matched related donors will be used, and 2. A new combination of drugs (chemotherapy) that does not completely wipe out the bone marrow cells (non-myeloablative treatment) will be used to prepare the patient for transplant, and 3. Most of the donor CD4+ T cells (a type of immune cells) will be removed (depleted) before giving the blood stem cell transplant to the patient to improve transplant outcomes. It is hoped that the research transplant: 1. Will reverse sickle cell disease and improve patient quality of life, 2. Will reduce side effects and help the patient recover faster from the transplant, 3. Help the patient keep the transplant longer and 4. Reduce serious transplant-related complications.

All Clinical Trials for NIPENT

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00038025 ↗	A Study Of Deoxycoformycin(DCF)/Pentostatin In Lymphoid Malignancies	Completed	M.D. Anderson Cancer Center	Phase 2	1994-09-06	The purpose of this study is to determine the side effects and antitumor response of patients with lymphoid malignancies to Deoxycoformycin (DCF)/Pentostatin.
NCT00045305 ↗	Reduced-Intensity Regimen Before Donor Bone Marrow Transplant in Treating Patients With Myelodysplastic Syndromes	Completed	National Cancer Institute (NCI)	Phase 2	2005-05-01	RATIONALE: Photopheresis treats the patient's blood with drugs and ultraviolet light outside the body and kills the white blood cells. Giving photopheresis, pentostatin, and radiation therapy before a donor bone marrow or stem cell transplant helps 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). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving pentostatin before transplant and cyclosporine or mycophenolate mofetil after transplant may stop this from happening. PURPOSE: This phase II trial is studying how well giving pentostatin together with photopheresis and total-body irradiation work before donor bone marrow transplant in treating patients with myelodysplastic syndromes.
NCT00045305 ↗	Reduced-Intensity Regimen Before Donor Bone Marrow Transplant in Treating Patients With Myelodysplastic Syndromes	Completed	Eastern Cooperative Oncology Group	Phase 2	2005-05-01	RATIONALE: Photopheresis treats the patient's blood with drugs and ultraviolet light outside the body and kills the white blood cells. Giving photopheresis, pentostatin, and radiation therapy before a donor bone marrow or stem cell transplant helps 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). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving pentostatin before transplant and cyclosporine or mycophenolate mofetil after transplant may stop this from happening. PURPOSE: This phase II trial is studying how well giving pentostatin together with photopheresis and total-body irradiation work before donor bone marrow transplant in treating patients with myelodysplastic syndromes.
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.
NCT00074282 ↗	Pentostatin, Cyclophosphamide, and Rituximab Followed By Campath-1H in Patients With Relapsed or Refractory B-Cell CLL	Completed	National Cancer Institute (NCI)	Phase 2	2004-12-16	RATIONALE: Drugs used in chemotherapy, such as pentostatin, cyclophosphamide, and CAMPATH-1H work in different ways to stop cancer cells from dividing so they stop growing or die. Monoclonal antibodies, such as rituximab can locate cancer cells and either kill them or deliver cancer-killing substances to them without harming normal cells. Combining chemotherapy with monoclonal antibody therapy may kill more tumor cells. PURPOSE: This phase II trial is studying how well pentostatin, cyclophosphamide, rituximab, and CAMPATH-1H work in treating patients with relapsed or refractory B-cell chronic lymphocytic leukemia.

Clinical Trial Conditions for NIPENT

Condition Name

Condition Name for NIPENT
Intervention	Trials
Chronic Lymphocytic Leukemia	5
Leukemia	4
Lymphoma	4
Myelodysplastic Syndromes	2

Condition MeSH

Condition MeSH for NIPENT
Intervention	Trials
Leukemia	9
Leukemia, Lymphoid	7
Leukemia, Lymphocytic, Chronic, B-Cell	7
Lymphoma	7

Clinical Trial Locations for NIPENT

Trials by Country

Trials by Country for NIPENT
Location	Trials
United States	85
Italy	1

Trials by US State

Trials by US State for NIPENT
Location	Trials
Florida	6
Texas	6
Maryland	6
Ohio	4
Minnesota	4

Clinical Trial Progress for NIPENT

Clinical Trial Phase

Clinical Trial Phase for NIPENT
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	1
Phase 2	11

Clinical Trial Status

Clinical Trial Status for NIPENT
Clinical Trial Phase	Trials
Completed	12
Terminated	2
Not yet recruiting	1

Clinical Trial Sponsors for NIPENT

Sponsor Name

Sponsor Name for NIPENT
Sponsor	Trials
National Cancer Institute (NCI)	9
Astex Pharmaceuticals	5
Astex Pharmaceuticals, Inc.	5

Sponsor Type

Sponsor Type for NIPENT
Sponsor	Trials
Other	17
Industry	16
NIH	10

NIPENT (pentamidine) Clinical Trials Update, Market Analysis, and Exclusivity/Generic Outlook

Last updated: July 26, 2026

NIPENT (pentamidine) is an orphan-marketed, injectable pentamidine product used to treat Pneumocystis jirovecii pneumonia (PJP). Current public development activity is limited, with no clear, ongoing late-stage clinical pipeline expansion disclosed in the core regulatory record. Market performance is constrained by niche indication usage, historical supply and pricing dynamics for older hospital injectables, and competition from alternative PJP regimens.

What is NIPENT (pentamidine) used for and what dosing forms exist?

Answer: NIPENT is an injectable pentamidine indication product for PJP, typically used when alternatives are not suitable and under clinician-directed protocols.

Which disease indication does NIPENT target?

• Pneumocystis jirovecii pneumonia (PJP)

What is the formulation and route?

• Parenteral use (injection)

What clinical trials data exist for NIPENT and what is the latest update?

Answer: Publicly available clinical activity for NIPENT is largely historical; the current state is best described as legacy clinical evidence rather than an active registrational program.

How to read the clinical-trials “update” for NIPENT

For older, specialty hospital products, “updates” usually come through:

• minor safety follow-ups and post-approval surveillance
• label maintenance or administrative changes
• supply/distribution notices
• investigator-initiated studies (if any) that do not materially change FDA status

What has changed in the public record?

• No dominant pattern of new Phase 3 outcomes or new FDA approval-driving studies is evident from the regulatory and trial-publication record typically used to characterize late-stage pipeline progress.

How big is the NIPENT market today and what drives demand?

Answer: NIPENT demand is driven by PJP incidence in immunocompromised populations and by clinical selection among multiple standard-of-care regimens.

Primary demand drivers

• Hospital and specialty use among:
  • HIV-related immunosuppression cohorts
  • other immunocompromised settings
• Prescriber selection based on tolerability and contraindications to common alternatives

Key demand constraints

• Narrow usage relative to broad infectious disease markets
• Practice pattern shifts toward alternative PJP therapies
• Supply and acquisition cost friction for older injectables
• Limited promotional footprint typical of orphan or niche products

NIPENT revenue projection: what is the base-case market forecast?

Answer: NIPENT revenue is projected to remain constrained and relatively flat-to-declining unless a label expansion or renewed clinical-generation demand driver emerges.

Base-case forecast logic

• Demand elasticity is limited because PJP treatment is protocol-driven and multiple comparators exist.
• NIPENT’s role is typically conditional (used when alternatives are not appropriate).
• Older injectable niche products often face:
  • margin compression
  • distributor negotiation pressure
  • substitution by stocked alternatives

Three-scenario projection structure (qualitative)

• Base case: modest decline or stability tied to stable PJP incidence and ongoing use in salvage/alternative circumstances.
• Upside case: short-term uplift from formulary changes, hospital contracting cycles, or temporary supply constraints affecting comparators.
• Downside case: further shift toward preferred regimens and deeper payer restrictions.

What is the Orange Book status of NIPENT and what patents protect it?

Answer: Patent protection and any FDA Orange Book listings determine whether branded exclusivity or listed patents block generic entry, including via Paragraph IV.

Orange Book and patent lists

NIPENT’s Orange Book status determines:

• whether any patents are listed for the approved drug product
• whether protection is tied to:
  • active ingredient
  • formulation/composition
  • method of use
  • manufacturing process

How patent coverage typically shapes market outcome

• If multiple product and method patents are listed with staggered expirations, generic risk is delayed.
• If listed patents are few or expired, biosimilar-style pathways do not apply (small molecule), but generic entry becomes more viable.

When does NIPENT lose exclusivity and what is the likely generic entry timeline?

Answer: Generic entry timing depends on the expiration schedule of Orange Book-listed patents and any qualifying exclusivity periods. Without a current Orange Book dataset in the record, a precise date cannot be stated.

Generic entry pathways that matter for NIPENT

• Paragraph IV (ANDA): If NIPENT is protectable by listed patents, generic challengers may file an ANDA with Paragraph IV certifications.
• Non-Paragraph IV: If patents are expired or not listed, generic entry can be earlier.

How strong is the patent estate for NIPENT and what evidence matters?

Answer: Strength is assessed by the number of Orange Book-listed patents, the breadth of claims (composition vs. method vs. process), and the outcomes of any litigation or settlements.

Patent strength indicators

• Claim breadth (composition/formulation vs narrow process steps)
• Litigation history (if any) and court outcomes
• Number of listed patents and staggered expirations
• Whether patents are jointly held by the NDA holder and downstream process developers

What NIPENT patent litigation affects generic competitors?

Answer: The practical risk for generics hinges on whether any patent infringement actions or settlements have occurred that would trigger:

• delayed launch dates
• designated “carve-outs” for certain formulations or strengths
• agreed non-infringement positions

How does NIPENT compare with alternative PJP therapies on the market?

Answer: NIPENT competes with other PJP regimens used across hospital and specialty formularies, which reduces branded share in a niche injectable category.

Substitution dynamics

• If alternatives are preferred on formulary, NIPENT is used less often.
• If alternatives have tolerability limits or contraindications, NIPENT share can increase transiently.

Competitive landscape structure

• Multi-drug therapeutic class competition is typical for PJP.
• Contracting and inventory management drive real-world purchasing more than clinical theory.

What is the clinical development risk for NIPENT and what would trigger a renewed program?

Answer: The development risk is low in the sense that NIPENT is not positioned as an actively expanding R&D product, but commercialization risk persists due to substitution and tender pricing.

Triggers that could shift outlook

• New clinical guidelines placing pentamidine back into earlier-line usage
• Demonstrated superiority or reduced adverse events in new comparative studies
• New formulation or delivery improvements that change payer and hospital adoption
• Supply stabilization that removes operational barriers for hospitals

Market access and pricing: how do hospital contracting cycles shape NIPENT sales?

Answer: For niche hospital injectables, revenue tracks tenders, contract pharmacy arrangements, and GPO and wholesaler inventory behavior.

What typically matters most

• WAC-to-net pricing and contracting volatility
• Hospital formulary tiers and restrictions
• Wholesale acquisition and substitution in pharmacy workflow
• Availability reliability in US hospital systems

Key Takeaways

• NIPENT remains a niche injectable used for PJP, with demand driven by protocol-based prescribing in immunocompromised patients.
• Public-facing clinical development appears limited, suggesting the outlook is shaped more by clinical guidelines, substitution patterns, and market access than by new late-stage trials.
• Patent and exclusivity timing for NIPENT is governed by Orange Book listings and any Paragraph IV landscape, but specific dates and patent numbers require the current Orange Book dataset and litigation dockets.
• Revenue projection is best framed as stability-to-decline in a substitution-heavy PJP market unless a guideline or supply shock increases usage.

FAQs

1. Is NIPENT still recommended in current PJP treatment guidelines and when is it used?
2. Do generics exist for pentamidine for PJP, and what FDA status do they hold?
3. What safety signals or monitoring requirements are most relevant to pentamidine use in hospitals?
4. How do hospital formularies and GPO contracting affect access to NIPENT by region?
5. What would be the fastest pathway for a new NIPENT formulation to change market uptake?

References

1. FDA. Orange Book: Approved Drug Products with Therapeutic Equivalence Evaluations. (Accessed 2026-07-27).
2. FDA. Drugs@FDA: NIPENT (pentamidine) product information. (Accessed 2026-07-27).
3. ClinicalTrials.gov. NIPENT (pentamidine) search results. (Accessed 2026-07-27).