Therapeutic Areas

The United Therapeutics product portfolio addresses the critical and unmet needs of patients with rare diseases and end-stage lung diseases.

With a foundational focus on vascular conditions, the majority of our therapies are concentrated in Pulmonary Arterial Hypertension (PAH; Group 1) and Pulmonary Hypertension-Associated with Interstitial Lung Disease (PH-ILD; Group 3).

Our goal is to reduce the progression of, and ultimately put an end to, end-stage organ disease through our organ manufacturing programs.

Our product offerings have also expanded into the field of pediatric oncology with a therapy approved to treat high-risk neuroblastoma as part of United Therapeutics’ commitment to improving the lives of those living with orphan diseases.

Pulmonary Arterial Hypertension (PAH)Group 1

Group 1 pulmonary hypertension is also known as pulmonary arterial hypertension (PAH). PAH is distinguished by high blood pressure that specifically occurs in the vessels that supply the lungs. This is distinct from more generalized hypertension which is characterized by high blood pressure in vessels throughout the body.

As part of United Therapeutics' commitment to advancing care for patients living with PAH we offer treatments with diverse administration options. United Therapeutics has four medications currently approved to treat PAH.

The United Therapeutics Corporation logo and The United by PH logo Learn more about PAH Learn more about our treprostinil franchise

Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD)Group 3

Interstitial lung disease (ILD) is a group of lung diseases that are characterized by marked scarring or fibrosis of the bronchioles and alveolar sacs within the lungs. Increased fibrotic tissue in ILD prevents oxygenation and free gas exchange between the pulmonary capillaries and alveolar sacs, and the condition can present with a wide range of symptoms, including shortness of breath with activity, labored breathing, and fatigue.

Group 3 pulmonary hypertension (PH) frequently complicates the course of patients with interstitial lung disease and is associated with worse functional status measured by exercise capacity, greater supplemental oxygen needs, decreased quality of life, and worse outcomes. PH is estimated to affect at least 15% of patients with early-stage ILD (approximately 30,000 PH-ILD patients in the United States) and may affect up to 86% of patients with more severe ILD.

Learn more about PH-ILD

Neuroblastoma

Neuroblastoma is a rare cancer that primarily affects children. Neuroblastoma occurs when immature nerve cells (also called neuroblasts) fail to normally develop into mature nerve cells. These immature nerve cells then multiply, leading to the growth of a cancerous mass of cells (tumor).

Neuroblastoma tumors can originate anywhere in the body but are often found in the abdomen or belly and usually involve the adrenal glands, which sit on top of the kidney. The chest, neck, hips, bone marrow, and pelvis are other sites that are commonly affected by neuroblastoma.

Once diagnosed, neuroblastoma can be further classified according to the level of risk (i.e., low, intermediate, or high-risk) it may pose to the patient. Risk classification is based on factors known to affect prognosis (i.e., chance of recovery) and the risk of the neuroblastoma returning after treatment (i.e., relapse). The factors that impact risk classification include age of diagnosis, stage of disease, and other characteristics of the tumor.

Learn more about Neuroblastoma

End-Stage Lung Disease

End-stage lung disease occurs when a chronic lung disease (e.g., COPD) reaches its most advanced state and severely compromises lung function. During end-stage lung disease, the function of the lungs may be reduced to 30% or less, and airflow in patients may be significantly limited. Consequently, patients with end-stage lung disease may not be able to complete even simple tasks (e.g., shower, cook).

Learn more about End-Stage Lung Disease