CLINICAL TRIALS PROFILE FOR PROMACTA KIT

All Clinical Trials for PROMACTA KIT

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00643929 ↗	LENS - Long-term Eltrombopag Observational Study	Completed	GlaxoSmithKline		2007-02-01	A long term observational ocular safety study in adults who have received study medication (either active drug or placebo) in a phase II or III clinical study evaluating eltrombopag. The study will follow subjects for 2.5 years following their last ocular assessment on their prior treatment study (regardless of the therapeutic indication) and will describe long-term ocular safety with respect to changes in the lenses over time from all subjects.
NCT00909363 ↗	Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients	Terminated	Novartis Pharmaceuticals	Phase 2	2009-06-01	The purpose of this project is to describe the pathophysiology of thrombocytopenia and bleeding in patients with Wiskott-Aldrich Syndrome (WAS) and determine the response to thrombopoietic agents in vitro and in vivo.
NCT00909363 ↗	Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients	Terminated	Weill Medical College of Cornell University	Phase 2	2009-06-01	The purpose of this project is to describe the pathophysiology of thrombocytopenia and bleeding in patients with Wiskott-Aldrich Syndrome (WAS) and determine the response to thrombopoietic agents in vitro and in vivo.
NCT00922883 ↗	A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia Patients	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 2	2009-05-29	Severe aplastic anemia (SAA) is a life-threatening blood disease which can be effectively treated with immunosuppressive drug regimens or allogeneic stem cell transplantation. However, 20-40% of patients without transplant options do not respond to immunosuppressive therapies, and have persistent severe cytopenias, requiring regular platelet transfusions, which are expensive and inconvenient, and are a risk for further serious bleeding complications. Thrombopoietin (TPO) is the principal endogenous regulator of platelet production and also stimulates hematopoietic stem and progenitor cells. A small molecule oral TPO-agonist, eltrombopag has been shown to increase platelets in healthy subjects and in patients with immune thrombocytopenic purpura (ITP), and received FDA approval in 2008 for the treatment of thrombocytopenia in ITP. This Phase 2, non-randomized pilot study of eltrombopag in aplastic anemia patients with immunosuppressive therapy refractory thrombocytopenia will test the safety and potential efficacy of eltrombopag treatment patients with refractory thrombocytopenia following immunosuppression for aplastic anemia. Subjects will initiate study medication at an oral dose of 50 mg/day, which will be increased up to 150 mg/day as clinically indicated to the lowest dose that maintains a stable platelet count 20,000/(micro)L above baseline while maximizing tolerability. Response will be assessed at 3-4 months. Platelet response is defined as platelet count increases to 20,000/L above baseline at three months. or stable platelet counts with transfusion independence for a minimum of 8 weeks. Erythroid response for subjects with a pretreatment hemoglobin of less than 9 g/dL will be defined as an increase in hemoglobin by greater than or equal to 1.5g/dL without packed red blood cell (PRBC) transfusion support, or a reduction in the units of transfusions by an absolute number of at least 4 PRBC transfusions for eight consecutive weeks compared with the pretreatment transfusion number in the previous 8 weeks. Neutrophil response will be defined in those with a pretreatment absolute neutrophil count (ANC) of less than 0.5 times 10(9)/L as at least a 100 percent increase or an absolute increase greater than 0.5 times 10(9)/L. Subjects with response at 3-4 months may continue study medication (extended access) until they meet an off study criteria. The primary objective is to assess the safety and efficacy of the oral thrombopoietin receptor agonist (TPO-R agonist) eltrombopag in aplastic anemia patients with immunosuppressive-therapy refractory thrombocytopenia. Secondary objectives include the analysis of the incidence and severity of bleeding episodes, and the impact on quality of life.
NCT00961064 ↗	A Pilot Study of a Thrombopoietin-Receptor Agonist, Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS)	Active, not recruiting	National Heart, Lung, and Blood Institute (NHLBI)	Phase 2	2009-07-24	Background: - Myelodysplastic syndromes (MDS) are bone marrow disorders characterized by anemia, neutropenia, and thrombocytopenia (low red blood cell, white blood cell, and platelet counts). Patients with MDS are at risk for symptomatic anemia, infection, and bleeding, as well as a risk of progression to acute leukemia. Standard treatments for MDS have significant relapse rates. MDS patients with thrombocytopenia who fail standard therapies require regular, expensive, and inconvenient platelet transfusions, and are at risk for further serious bleeding complications. - Eltrombopag is a drug designed to mimic the protein thrombopoietin, which causes the body to make more platelets. Eltrombopag has been able to increase platelet counts in healthy volunteers and in patients with chronic ITP (a disease where patients destroy their own platelets very rapidly and thus develop thrombocytopenia), but researchers do not know if the drug can increase platelet counts in patients with MDS. Objectives: - To find out whether eltrombopag can improve platelet counts in patients with MDS. - To determine whether eltrombopag is safe for patients with MDS. Eligibility: - Patients 18 years of age and older who have consistently low blood platelet counts related to MDS that has not responded to conventional treatment. - Platelet count ≤ 30,000/μL or platelet-transfusion-dependence (requiring at least 4 platelet transfusions in the 8 weeks prior to study entry); OR hemoglobin less than 9.0 gr/dL or red cell transfusion-dependence (requiring at least 4 units of PRBCs in the eight weeks prior to study entry) OR ANC≤500 Design: - Treatment with eltrombopag tablets once per day for 16-20 weeks. - Participants will be monitored closely throughout the initial treatment, with weekly blood tests and separate evaluations at the National Institutes of Health (NIH) treatment center every 4 weeks. Bone marrow biopsies may be conducted to check for abnormalities in bone marrow. - If patients show signs of improved platelet counts after 90 days, treatment will continue with additional doses of eltrombopag. - Patients who discontinue taking eltrombopag will be evaluated at the NIH treatment center 4 weeks after ending treatment, and again 6 months after ending treatment to check for potential side effects.
NCT00996216 ↗	Clinical Trial for Non-responders Who Previously Participated in Eltrombopag Studies TPL 103922 or TPL 108390	Completed	GlaxoSmithKline	Phase 3	2009-09-01	The purpose of this study is to test the safety and tolerability of eltrombopag when used to increase and maintain platelet count. Platelet count to be maintained at a level sufficient to facilitate initiation of antiviral therapy, to minimize antiviral therapy dose reductions, and to avoid permanent discontinuation of antiviral therapy.

Clinical Trial Conditions for PROMACTA KIT

Condition Name

Condition Name for PROMACTA KIT
Intervention	Trials
Thrombocytopenia	9
Leukemia	4
Hepatitis C	3
Adult Acute Myeloid Leukemia With Del(5q)	2

Condition MeSH

Condition MeSH for PROMACTA KIT
Intervention	Trials
Thrombocytopenia	16
Leukemia	8
Preleukemia	6
Myelodysplastic Syndromes	6

Clinical Trial Locations for PROMACTA KIT

Trials by Country

Trials by Country for PROMACTA KIT
Location	Trials
United States	52
Italy	13
Germany	10
Canada	10
Brazil	8

Trials by US State

Trials by US State for PROMACTA KIT
Location	Trials
Texas	7
New York	6
Maryland	5
California	4
North Carolina	3

Clinical Trial Progress for PROMACTA KIT

Clinical Trial Phase

Clinical Trial Phase for PROMACTA KIT
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	3
Phase 2/Phase 3	2

Clinical Trial Status

Clinical Trial Status for PROMACTA KIT
Clinical Trial Phase	Trials
Completed	11
Terminated	8
Active, not recruiting	6

Clinical Trial Sponsors for PROMACTA KIT

Sponsor Name

Sponsor Name for PROMACTA KIT
Sponsor	Trials
GlaxoSmithKline	15
M.D. Anderson Cancer Center	6
Novartis Pharmaceuticals	5

Sponsor Type

Sponsor Type for PROMACTA KIT
Sponsor	Trials
Other	29
Industry	26
NIH	9

Promacta Kit clinical trials update, market analysis and 2026 to 2035 projection

Last updated: May 21, 2026

Promacta Kit (eltrombopag) market outlook is driven by chronic immune thrombocytopenia (ITP), chronic liver disease-associated thrombocytopenia, and ongoing competition from generic eltrombopag and product substitution. Near-term growth depends on formulary access and label expansion execution rather than new-to-world efficacy. The clinical-trial pipeline shows incremental studies across adult and pediatric ITP and real-world validation, while the commercial trajectory increasingly reflects managed care pressure and pricing dynamics.

What is Promacta Kit (eltrombopag) and how is it used clinically?

Featured snippet: Promacta Kit is eltrombopag therapy used to treat thrombocytopenia in immune thrombocytopenia (ITP) and to raise platelet counts to allow initiation and maintenance of interferon-based hepatitis C treatment and in other thrombocytopenia indications where platelet augmentation is needed.

Which indications drive revenue?

Key label areas (US/EU) include:

• Adult chronic immune thrombocytopenia (chronic ITP), including after insufficient response to corticosteroids, immunoglobulins, or splenectomy.
• Pediatric chronic ITP.
• Thrombocytopenia associated with chronic hepatitis C in historical contexts tied to interferon regimens (the role has narrowed as hepatitis C standard of care moved to DAAs).

Dosing and administration factors that impact uptake

• Oral eltrombopag with platelet monitoring schedule.
• Dose titration to reach and maintain target platelet counts.
• Discontinuation criteria to mitigate excessive platelet elevations.

What is the current clinical trials update for Promacta (eltrombopag)?

Featured snippet: Active development has shifted toward label refinement, long-term safety/registry studies, special populations, and practical endpoints like platelet normalization durability and treatment persistence.

How trial design trends map to clinical practice

Clinical trial updates for eltrombopag broadly reflect:

• Platelet response endpoints (overall response rate, durable response, time to loss of response).
• Safety endpoints focused on thromboembolic events, liver enzyme elevations, and marrow or reticulin-related signals.
• Real-world studies and observational cohorts assessing persistence and discontinuation drivers.

Recent themes seen across eltrombopag development

• Pediatric continuation and growth/safety monitoring.
• Retreatment and discontinuation strategy in chronic ITP.
• Comparative effectiveness in steroid-sparing pathways and sequencing with other ITP agents (TPO-RAs, Syk inhibitors, monoclonal antibodies).

Which ongoing studies are most likely to change Promacta market adoption?

Featured snippet: Studies that improve durability of platelet response, clarify discontinuation windows, and quantify real-world persistence in chronic ITP can drive formulary confidence and payer acceptance.

High-leverage study types

• Long-term extension studies that tighten risk-benefit around sustained therapy.
• Studies in patients previously exposed to other ITP lines, including sequencing insights.
• Studies with standardized platelet monitoring and dose adjustment algorithms.

How strong is the patent and exclusivity position behind Promacta (eltrombopag)?

Featured snippet: Promacta’s exclusivity is largely anchored in historical patent and regulatory exclusivity regimes rather than new compound exclusivity today, with competition risk tied to generic and authorized generic entry.

What this means for market dynamics

• Once generic coverage expands across key dosage strengths and routes, price competition compresses margins.
• Specialty pharmacy distribution and contracting determine realized net price more than headline list price.

What is the Orange Book status of eltrombopag-related products?

Featured snippet: The Orange Book tracks approved eltrombopag products and associated patents by submission type and expiration; the commercial impact is determined by which patents remain listed and which are challenged via Paragraph IV.

Market-relevant takeaways

• Patent-expiration timing and whether patents are actively enforced shape launch timing for generics.
• For business planning, the key question is not “is eltrombopag generic?” but “which strengths are contracting-favored and who controls distribution for those strengths.”

How do clinical outcomes compare between Promacta and alternative ITP therapies?

Featured snippet: In chronic ITP, eltrombopag competes primarily in the TPO-RA class, where response rates and durability are generally comparable, and differences that matter are administration burden, onset, discontinuation feasibility, and safety management.

Competitive set for chronic ITP

• Other TPO receptor agonists: avatrombopag, romiplostim, and related strategies depending on region.
• Non-TPO RA agents used in lines of therapy: Syk inhibitors (eg, fostamatinib), BTK or other targeted approaches where approved, and rescue strategies.

What typically decides formulary placement

• Payer preference for dosing frequency and monitoring burden.
• Evidence in post–splenectomy and multi-refractory populations.
• Real-world persistence and safety management performance.

What is the current market size for eltrombopag/Promacta and what are the growth drivers?

Featured snippet: The eltrombopag market is mature and increasingly influenced by generic penetration and net-price declines, while demand persists from chronic ITP incidence and chronicity.

Core demand drivers

• Ongoing incident cases and relapsing nature of chronic ITP.
• Continued need for second-line and later-line platelet raising with manageable tolerability.
• Stable clinical recognition of TPO-RAs in platelet management plans.

Primary headwinds

• Generic competition and shelf access by lower-cost entrants.
• Payer and PBM contracting pressure toward lowest net-cost options within TPO-RA class.
• Shift in hepatitis C therapeutic landscape reduces relevance of interferon-tied indications.

How does Promacta pricing and contracting pressure affect revenue projections?

Featured snippet: Revenue projections must model net price compression from generic substitution and contract renegotiations, with specialty channel demand transferring to the lowest net-cost covered product.

Operational levers that can offset erosion

• Managed entry agreements with key PBMs.
• Patient assistance and adherence programs that maintain treatment continuity.
• Strength-specific channel focus where generics may have less market access.

Promacta Kit forecast 2026 to 2035: base, downside, and upside scenarios

Featured snippet: Without a material label expansion, Promacta’s trajectory is most sensitive to net price and the pace of generic substitution.

Projection framework (what drives the curve)

• Volume: chronic ITP-treated population and treatment persistence.
• Price: net realized price after rebates and PBM contracting.
• Mix: adult versus pediatric share, line-of-therapy share, and concomitant liver disease mix.
• Competitive access: formulary coverage of generics and alternative TPO-RAs.

Scenario table

The following is a scenario structure for revenue planning (not a commitment of realized figures):

Year	Base case (moderate erosion)	Downside (faster substitution)	Upside (stable net price + persistence)
2026	Low single-digit net erosion	Mid single-digit erosion	Flat-to-low growth
2028	Continued price compression	Higher erosion as contracts flip	Mild erosion offset by persistence
2030	Mature market dynamics	Marked volume and price loss	Better-than-market channel retention
2035	Stabilizes at low-growth niche	Competitive saturation	Niche stabilization only

What generic entry risks exist for Promacta (eltrombopag) by strength and route?

Featured snippet: Generic entry risk is strongest where patent listings expire and where manufacturing capacity enables rapid coverage across key strengths.

What to model

• Time-to-launch by strength (some strengths may launch earlier based on ANDA status and manufacturing readiness).
• Coverage breadth and channel uptake (specialty pharmacy contracts decide realized demand).
• Any authorized generic strategy by challengers.

What patent litigation affects Promacta’s competitive landscape?

Featured snippet: Litigation is primarily relevant to whether specific generic products launch at a given time and whether additional design-around IP persists.

Business impact channels

• Settlement agreements that delay launch can support higher net pricing for branded product.
• Final court determinations affect launch timing by specific ANDA product(s).

What biosimilar risk applies to Promacta?

Featured snippet: Biosimilar risk is not directly applicable because eltrombopag is a small-molecule drug, not a biologic.

What matters instead

• ANDA generic small-molecule substitution.
• Fixed-dose combination substitutes if any exist in practice.
• TPO-RA class competitive switching (especially where payer prefers a particular agent).

Which countries have the highest commercial exposure for eltrombopag/Promacta?

Featured snippet: Commercial exposure is highest in major markets where chronic ITP treatment volume is concentrated and reimbursement supports TPO-RA adoption.

Regional factors

• US: payer contracting and generic penetration pace.
• EU5 (Germany, France, UK, Spain, Italy): centralized and country-specific reimbursement decisions.
• Japan and other regulated markets: slower substitution dynamics can temporarily support pricing but eventual generic availability still compresses net revenue.

How does Promacta compare with other ITP TPO-RAs on marketability and payer fit?

Featured snippet: Within TPO-RAs, marketability depends on payer preference, dosing convenience, safety management protocols, and formulary history.

Practical differentiators that influence adoption

• Administration schedule and ease of titration.
• Patient monitoring burden alignment with clinic workflows.
• Rate of platelet response and durability patterns in refractory subgroups.

Key takeaways

• Promacta’s market trajectory is mature and increasingly governed by generic substitution and net price contracting rather than major incremental clinical breakthroughs.
• Clinical trial updates increasingly focus on long-term durability, special populations, and real-world persistence, which influence payer and formulary confidence.
• Forecasts from 2026 to 2035 hinge on three variables: net realized price erosion speed, persistence/continuation under payer pressure, and competitive channel access versus other ITP TPO-RAs.

FAQs

1. What is the main mechanism of action of eltrombopag in chronic ITP?
2. How do platelet response endpoints in eltrombopag trials translate into payer coverage decisions?
3. Which ITP subpopulations show the highest persistence on eltrombopag in real-world cohorts?
4. How should forecast models incorporate strength-level generic launch timing for eltrombopag?
5. What safety monitoring elements most affect treatment continuity for Promacta users?

References

1. FDA. Drug Approval Package: Promacta (eltrombopag). U.S. Food and Drug Administration.
2. FDA. Drugs@FDA: Promacta (eltrombopag) information. U.S. Food and Drug Administration.
3. FDA. Orange Book: Approved Drug Products with Therapeutic Equivalence Evaluations. U.S. Food and Drug Administration.