If you’re living with pulmonary arterial hypertension (PAH), you may already know about the treatments available today. However, you might not be aware that new and promising options are in the works.

Researchers have developed medications that don’t just manage symptoms — they target the root cause of PAH. This article explores some of the most exciting new treatments available now and those underway. If you have questions after reading, talk to your pulmonologist (lung specialist) for more information.

Current Treatment of Pulmonary Arterial Hypertension: Oral, Intravenous, and Other Options

Most current PAH treatments work by widening blood vessels or reducing fluid in the body. This makes it easier for the heart to pump blood through the lungs. Standard treatments for PAH and pulmonary hypertension (PH) include:

• Medicines that help the heart beat stronger
• Diuretics (water pills)
• Blood thinners
• Supplemental oxygen
• Calcium channel blockers
• Medicines that improve blood flow (like phosphodiesterase 5 inhibitors)
• Medicines that open blood vessels (like endothelin receptor antagonists)
• Medicines that lower pressure in the lungs (like soluble guanylate cyclase stimulators)
• Vasodilators, which relax blood vessels (like prostacyclin analogs)

PAH medications may be taken orally (by mouth), inhaled (breathed in), injected intravenously (delivered into a vein), or injected subcutaneously (delivered under the skin).

Medications can target the body’s main pathways affected by PAH. You can think of these pathways as different roads that medicines travel to reach their destination — helping your heart and lungs work better so you can breathe more easily. These treatments can ease some symptoms, like shortness of breath and fatigue, but they don’t cure pulmonary arterial hypertension.

Treatment plans often involve combinations of medications, guided by a pulmonologist or cardiologist (heart specialist). However, not every treatment works for every person. Some medications may cause side effects, and IV drugs may be difficult to manage long term.

Recent Advances in PAH Treatment

PAH is often linked to a combination of genetics, growth factors (proteins that affect how cells grow and work), inflammation, hormones, and even iron levels. By studying these pathways, scientists are developing treatments that target the root causes of PAH — not just the symptoms.

To help explain these new therapies, we’ve grouped them by how they work in the body. Each one is being studied as an add-on to standard PAH treatment.

1. Tyrosine Kinase Inhibitors

Tyrosine kinase inhibitors (TKIs) help control cell growth. These drugs are often used to treat cancer by blocking the growth of harmful cells. Researchers are now testing TKIs to see if they could help treat PAH.

Imatinib

Imatinib is an oral TKI approved by the U.S. Food and Drug Administration (FDA) for several types of cancer. Early studies of the pill form in PAH showed some benefit but also caused serious side effects.

In a small study, a daily 200-milligram dose of oral imatinib was well tolerated, with most people reporting only mild nausea. The treatment lowered both lung blood pressure and total pulmonary resistance (the force required by the heart to pump blood through the lungs).

Researchers also studied an inhaled version of the drug, but they found that it didn’t improve pulmonary resistance or six-minute walk distance in people with PAH.

Seralutinib

Researchers are testing seralutinib, another inhaled TKI, in clinical trials — studies that test a potential therapy’s safety and effectiveness in humans. In animal studies, seralutinib reversed some of the changes PAH caused in blood vessels. Clinical trial results are expected in 2026.

2. Bone Morphogenetic Protein Signaling Modulators

Bone morphogenetic proteins (BMPs) were first known to help bones grow and heal. Scientists have since learned that BMPs also play an important role in many parts of the body, including the lungs. In PAH, the BMP signaling pathway doesn’t work properly, which can cause the walls of lung blood vessels to become thick and stiff. BMP modulators can help balance cell growth and keep these blood vessels healthier and more flexible.

In PAH, the BMP signaling pathway doesn’t work properly, which can cause the walls of lung blood vessels to become thick and stiff.

Sotatercept

The FDA approved sotatercept (Winrevair) in March 2024 to treat PAH. This drug works by restoring balance to growth signals in the blood vessels, which helps prevent thickening in the pulmonary arteries and supports healthier blood flow to the lungs. When combined with other therapies, sotatercept has been shown to improve blood flow and increase exercise capacity.

More recent research showed that adding sotatercept to standard treatment may improve survival, or how long someone lives, with World Health Organization (WHO) functional class 3 or 4 PAH. Compared to those who took a placebo (inactive substance), people who took sotatercept had a lower risk of death, were less likely to need a lung transplant, and had fewer hospital stays lasting longer than 24 hours due to worsening PAH.

Sotatercept isn’t right for everyone and can cause side effects. However, because it works differently from other PAH treatments, it offers a new way to target the underlying cause of the disease.

Elafin

Elafin is a compound that blocks elastase — an enzyme that breaks down elastin, a protein that keeps lung blood vessels strong and flexible. People with PAH tend to have higher levels of elastase, which can lead to stiff, thickened blood vessels. By blocking elastase, elafin may help protect the blood vessels and keep them working better.

In an early clinical study, elafin was shown to be safe and well tolerated, causing no serious side effects. A phase 2 clinical trial on people with PAH is expected to begin in 2026.

Tacrolimus

Tacrolimus, a medication that reduces inflammation, is FDA-approved to help prevent organ rejection after transplant. Since inflammation can stiffen blood vessels in the lungs, researchers are exploring whether tacrolimus could help treat PAH.

A small study tested low-dose tacrolimus over 16 weeks in people with PAH. The drug was found to be safe, but it didn’t lead to improvements in symptoms, ability to exercise, heart function, or lab markers related to heart failure.

3. Bromodomain Inhibitors

Bromodomain proteins (BRDs) help the body read and follow genetic instructions. When these proteins don’t work properly, they can trigger harmful changes in cells. Bromodomain inhibitors are being studied to block these changes, with the hope that they may help prevent heart and lung damage in conditions like PAH.

Apabetalone

Apabetalone blocks a bromodomain protein called BRD4, which can lead to swelling and scarring in PAH. In a small study, apabetalone improved pulmonary resistance and cardiac output. These promising results need to be confirmed by larger studies.

4. Hormone-Targeted Therapy

Females are at higher risk of PAH, according to the journal Comprehensive Physiology. This knowledge has led researchers to look into therapies that target estrogen, a hormone involved in reproductive health, as a possible PAH treatment.

Researchers are studying therapies that target estrogen as a possible PAH treatment.

Tamoxifen

Tamoxifen is FDA-approved to treat estrogen receptor-positive breast cancer. Also available as a generic, tamoxifen is well studied for its safety and effectiveness. In 2023, researchers completed a study to see if this antiestrogen therapy could help treat PAH. As of November 2025, the results had not yet been published.

Anastrozole

Anastrozole, another FDA-approved drug for estrogen receptor-positive breast cancer, has also been studied in people with PAH. In 2024, a large study concluded that anastrozole was safe — but, compared to a placebo (inactive substance), it didn’t significantly help participants walk farther (a common measure of PAH treatment success).

The Future of Treatments for Pulmonary Arterial Hypertension

While the research continues, many people living with PAH are eager to know what the future holds. A myPHteam member asked, “Has anyone heard of any new clinical trials coming out that may have the ability to cure this disease?”

Clinical trials play a key role in developing new treatments. By joining a clinical trial, you can help researchers learn more about PAH, and you might be able to try new treatments before they’re available to everyone. If you’re interested in a clinical trial, talk with your doctor to see if it might be a good fit for you.

In the meantime, keep your healthcare provider updated about how your current treatment is working and any side effects you’re experiencing. They can help you adjust your treatment plan to better manage your PAH and symptoms.

Join the Conversation

On myPHteam, people share their experiences with pulmonary hypertension, get advice, and find support from others who understand.

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