Single Ventricle Heart Disease Pipeline Analysis Report 2025

Single ventricle is a rare yet complicated heart condition, with a birth incidence of roughly 4-8 per 10,000, it accounts for 7.7% of congenital heart disease identified in children. Most patients will have neonatal cyanosis when they first arrive, but prenatal ultrasonography is now able to identify more of them. The single ventricle heart disease pipeline analysis by the publisher focuses on various treatment options for this disease. Recent developments include small molecule and biologics. Increasing research, improved diagnostics, and innovative treatments are expected to fuel the growth of single ventricle heart disease drug candidates in the coming years.

Report Coverage

The Single Ventricle Heart Disease Pipeline Analysis Report by the publisher gives comprehensive insights into single ventricle heart disease therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for Single Ventricle Heart Disease therapeutics. The single ventricle heart disease report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The single ventricle heart disease pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with single ventricle heart disease treatment guidelines to ensure optimal care practices.

The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to single ventricle heart disease.

Single Ventricle Heart Disease Pipeline Outlook

Single ventricle heart disease (SVHD) is a rare congenital heart condition where one of the heart’s two pumping chambers (ventricles) is underdeveloped or absent. This severely limits the heart’s ability to circulate blood efficiently, often requiring infants to undergo a series of surgeries to reroute blood flow. SVHD typically includes conditions like hypoplastic left heart syndrome or tricuspid atresia, and is associated with significant long-term health challenges, including exercise intolerance, heart failure, and arrhythmias.

SVHD is managed through staged surgical interventions, namely, Norwood, Glenn, and Fontan procedures. These procedures create alternative circulatory pathways. Post-surgery, ongoing treatment includes medications to support heart function, manage symptoms, and improve quality of life. Emerging pharmacologic therapies aim to enhance exercise capacity, cardiac output, and long-term outcomes in patients living with Fontan physiology. For example, in 2023, Mezzion Pharma Co. Ltd. submitted a New Drug Application (NDA) to the US FDA for Udenafil, a PDE5 inhibitor, for adolescents with Fontan physiology. This makes Udenafil a leading investigational therapy targeting long-term complications of single ventricle heart disease.

Single Ventricle Heart Disease Epidemiology

Single ventricles are very uncommon. 5 kids out of 10,000 are born with only one ventricle. The number of people with single ventricle heart disease who have Fontan circulation has increased to about 700,000 worldwide, with about half of them being older than 18, thanks to notable improvements in survival over the previous 40 years.

Single Ventricle Heart Disease - Pipeline Therapeutic Assessment

This section of the report covers the analysis of single ventricle heart disease drug candidates based on several segmentations, including:

By Phase

• Late-Stage Products (Phase 3 and Phase 4)
• Mid-Stage Products (Phase 2)
• Preclinical and Discovery Stage Products

By Drug Class

• Small Molecules
• Biologics

By Route of Administration

• Oral
• Parenteral
• Others

Single Ventricle Heart Disease - Pipeline Assessment Segmentation, By Phases

The report covers phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to analysis, phase III covers a major share of the total single ventricle heart disease clinical trials.

In the single ventricle heart disease pipeline, most candidates are in Phase III with 40% of the projects, followed by 20% in Phase I and 20% in Phase IV. Additionally, 20% are in Early Phase I, demonstrating a broad spectrum of development stages and diverse progress toward potential treatments.

Single Ventricle Heart Disease - Pipeline Assessment Segmentation, By Drug Classes

The drug molecule categories covered under the single ventricle heart disease pipeline analysis include small molecules and biologics. The single ventricle heart disease report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for single ventricle heart disease.

Particularly for individuals with Fontan physiology, soluble guanylate cyclase (sGC) stimulators are becoming new therapeutic medicines in the pipeline for single ventricle heart disease (SVHD). The nitric oxide (NO)-sGC-cyclic GMP (cGMP) signaling cascade, which is frequently dysregulated in SVHD, is enhanced by these tiny compounds, such as vericiguat. These drugs increase cardiac efficiency, decrease ventricular afterload, and enhance systemic and pulmonary vasodilation by directly activating sGC regardless of no availability. By addressing important physiological deficiencies that are not being addressed by current surgical or supportive care approaches, their dual action shows promise in enhancing exercise capacity, oxygen delivery, and long-term heart function in post-Fontan patients.

Single Ventricle Heart Disease Clinical Trials - Key Players

The report for the single ventricle heart disease pipeline analysis covers the profile of key companies involved in clinical trials and their drugs under development. Below is the list of a few players involved in single ventricle heart disease clinical trials:

• Merck Sharp & Dohme LLC
• Mezzion Pharma Co. Ltd
• Regeneron Pharmaceuticals

Single Ventricle Heart Disease - Emerging Drugs Profile

This section covers the detailed analysis of each drug under multiple phases, including phase II, phase III, phase IV, and emerging drugs for single ventricle heart disease. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of single ventricle heart disease drug candidates.

Drug: Vericiguat

The experimental small molecule Vericiguat is under development by Merck Sharp & Dohme LLC to treat single ventricle heart disease. It enhances the nitric oxide (NO)-sGC-cyclic GMP (cGMP) signaling pathway, which is essential for cardiovascular control, by acting as a soluble guanylate cyclase (sGC) stimulator. Vericiguat stimulates sGC directly and raises intracellular cGMP levels, which improves tissue perfusion, myocardial function, and vasodilation. The medication's effectiveness, safety, and effect on cardiac function in patients with single ventricle physiology, a complicated congenital heart abnormality that frequently results in heart failure are being assessed clinically. This finding offers a possible expansion into juvenile and congenital cardiology settings, building on Vericiguat's previous approval for heart failure with reduced ejection fraction (HFrEF).

Drug: Udenafil

Mezzion Pharma Co. Ltd. created the phosphodiesterase type 5 (PDE5) inhibitor Udenafil, which is being tested for the treatment of single ventricle heart disease, especially in young adults and adolescents who have had the Fontan operation. By preventing the breakdown of cyclic guanosine monophosphate (cGMP), udenafil improves the nitric oxide-cGMP pathway, resulting in vasodilation, decreased pulmonary vascular resistance, and increased cardiac output. Exercise intolerance and increasing heart failure are two long-term issues linked to Fontan physiology that the medication attempts to treat. The Fontan Udenafil Exercise Longitudinal (FUEL) experiment examined udenafil and showed promise in enhancing heart function and exercise capacity. Confirming its effectiveness, safety, and quality-of-life enhancements in this high-risk population with congenital cardiac disease is the goal of the current development effort.

Reasons To Buy This Report

The single ventricle heart disease drug pipeline insight report provides a strategic overview of the latest and future landscape of treatments for single ventricle heart disease. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into single ventricle heart disease collaborations, regulatory environments, and potential growth opportunities.

Key Questions Answered in the Single Ventricle Heart Disease Pipeline Analysis Report

• Which companies/institutions are leading the single ventricle heart disease drug development?
• What is the efficacy and safety profile of single ventricle heart disease pipeline drugs?
• Which company is leading the single ventricle heart disease pipeline development activities?
• What is the current single ventricle heart disease commercial assessment?
• What are the opportunities and challenges present in the single ventricle heart disease pipeline landscape?
• Which company is conducting major trials for single ventricle heart disease drugs?
• Which companies/institutions are involved in single ventricle heart disease collaborations aimed at providing enhanced therapeutic alternatives for patients?
• What are the geographies covered for clinical trials in single ventricle heart disease?

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