CLINICAL TRIALS PROFILE FOR PROMACTA

All Clinical Trials for PROMACTA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
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.
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.

Clinical Trial Conditions for PROMACTA

Condition Name

Condition Name for PROMACTA
Intervention	Trials
Thrombocytopenia	9
Leukemia	4
Hepatitis C	3
Adult Acute Myeloblastic Leukemia With Maturation (M2)	2

Condition MeSH

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

Clinical Trial Locations for PROMACTA

Trials by Country

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−

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

Trials by US State

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−

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

Clinical Trial Progress for PROMACTA

Clinical Trial Phase

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

Clinical Trial Status

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

Clinical Trial Sponsors for PROMACTA

Sponsor Name

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

Sponsor Type

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

Promacta: Clinical Trials, Market Analysis, and Projections

Introduction to Promacta

Promacta, also known as eltrombopag, is a small-molecule thrombopoietin (TPO) receptor agonist used to treat low platelet levels in patients with chronic immune thrombocytopenia (ITP) and other blood disorders. Here, we will delve into the clinical trials, market analysis, and future projections for this drug.

Clinical Trials Update

EXTEND Study

One of the significant clinical trials for Promacta is the EXTEND study, an open-label extension of four previous trials. This study enrolled 302 adults with chronic/persistent ITP who had received prior therapy for their condition. The key findings include:

• Efficacy: Median platelet counts were elevated to ≥50×10^9/L within two weeks of Promacta treatment and maintained for more than four years. Nearly 70% of patients maintained platelet counts of ≥30×10^9/L without rescue therapy, reducing the overall risk of bleeding[1].
• Reduction in Concomitant Medications: More than one-third of patients were able to permanently stop one or more concomitant ITP medications, including corticosteroids, danazol, and azathioprine[1].
• Safety Profile: The study established a long-term safety profile for Promacta, with common adverse events including headache, nasopharyngitis, and upper respiratory tract infection. Thromboembolic events were observed in 6% of patients[1].

Other Clinical Considerations

In addition to the EXTEND study, Promacta has been evaluated in various other clinical settings. For instance, a trial combining Promacta with azacitidine in patients with chronic hepatitis C was terminated due to lack of efficacy and safety concerns, including an increased risk of progression to acute myeloid leukemia (AML)[4].

Mechanism of Action and Dosage

Promacta works by interacting with the transmembrane domain of the human TPO-receptor, initiating signaling cascades that induce proliferation and differentiation of megakaryocytes from bone marrow progenitor cells. This leads to an increase in platelet production.

• Dosage: The drug is initiated at 50 mg once daily for most adult and pediatric patients 6 years and older, and at 25 mg once daily for pediatric patients aged 1-5 years. Dose adjustments are necessary for patients with hepatic impairment and some patients of East-/Southeast Asian ancestry to maintain a platelet count greater than or equal to 50 × 10^9/L[5].

Market Analysis

Current Market Size and Share

The global market for ITP treatments, including Promacta, is substantial and growing. In 2023, the total market size in the US for ITP was estimated to be nearly USD 2.80 billion. Promacta, along with Nplate/Romiplate, captured a significant share of this market, with Promacta being one of the leading therapies[3].

Regional Market Insights

• US and EU: The US and EU4 (Germany, France, Italy, and Spain) along with the UK are key markets for Promacta. The UK holds the highest market share in the EU4 and UK region[3].
• Japan: The Japanese market for ITP treatments, including Promacta, was estimated to be nearly USD 300 million in 2023[3].

Market Drivers and Trends

The ITP market is driven by several factors:

• Emerging Therapies: The introduction of novel therapies, including biologics, small molecules, and targeted treatments, is propelling market growth[3].
• Increased Awareness and Diagnostic Capabilities: Enhanced awareness and improved diagnostic capabilities are contributing to the expansion of the ITP market[3].
• Regulatory Milestones: Ongoing regulatory approvals and label expansions for current therapies are also driving market growth[3].

Market Projections

Forecasted Growth

The global Promacta market is expected to grow significantly from 2025 to 2032. Here are some key projections:

• Revenue Growth: The market is forecasted to experience revenue growth, driven by the increasing adoption of Promacta and other ITP treatments[2][5].
• Regional Growth: The US, EU4, UK, and Japan are expected to remain key markets, with the UK and Japan showing significant growth potential[3][5].

Competitive Landscape

Promacta faces competition from other ITP treatments such as:

• Tavalisse/Tavlesse (fostamatinib disodium hexahydrate): Marketed by Rigel Pharmaceuticals, Kissei Pharmaceutical, and Grifols[3].
• Doptelet (avatrombopag): Marketed by Sobi (Dova Pharmaceuticals) and Asahi Kasei Pharma[3].
• Emerging Drugs: Rilzabrutinib (PRN-1008) by Sanofi/Principia Biopharma and VAY736 (Ianalumab) by Novartis are among the emerging therapies in the ITP market[3].

Key Takeaways

• Clinical Efficacy: Promacta has demonstrated long-term efficacy in maintaining platelet counts and reducing bleeding risks in patients with chronic ITP.
• Market Size and Growth: The global ITP market, including Promacta, is substantial and expected to grow driven by emerging therapies and increased awareness.
• Regional Insights: The US, EU4, UK, and Japan are key markets, with significant growth potential in these regions.
• Competitive Landscape: Promacta competes with other ITP treatments, but its long-term safety and efficacy profile make it a trusted option.

FAQs

What is Promacta used for?

Promacta (eltrombopag) is used to treat low platelet levels in patients with chronic immune thrombocytopenia (ITP) and other blood disorders.

What are the common side effects of Promacta?

Common side effects include headache, nasopharyngitis, and upper respiratory tract infection. Thromboembolic events can also occur in some patients[1].

How does Promacta work?

Promacta works by interacting with the TPO-receptor, initiating signaling cascades that induce proliferation and differentiation of megakaryocytes, leading to an increase in platelet production[5].

What is the current market size for ITP treatments including Promacta?

In 2023, the total market size in the US for ITP was estimated to be nearly USD 2.80 billion, with Promacta capturing a significant share[3].

What are the emerging trends in the ITP market?

Emerging trends include the introduction of novel therapies such as biologics, small molecules, and targeted treatments, along with increased awareness and improved diagnostic capabilities[3].

What is the forecasted growth for the Promacta market?

The Promacta market is expected to grow significantly from 2025 to 2032, driven by increasing adoption and regulatory milestones[2][5].

Sources

1. Novartis Press Release: "Novartis drug Promacta® shows long-term disease control for chronic/persistent immune thrombocytopenia (ITP)" - Oct 18, 2017.
2. Cognitive Market Research: "Promacta Market Report 2024 (Global Edition)" - Oct 21, 2022.
3. GlobeNewswire: "Immune Thrombocytopenia (ITP) Industry Analysis and Forecast 2020-2034" - Dec 2, 2024.
4. FDA Label: "Promacta - accessdata.fda.gov" - 2023.
5. DelveInsight Report: "PROMACTA Drug Insight and Market Forecast - 2032" - Nov 1, 2023.