Dear friends,

Pulmonary hypertension treatment has come a long way since PHA’s founding in 1991. In the past week, the Food and Drug Administration approved the 15th and 16th treatments for pulmonary arterial hypertension.

Last Friday, the FDA approved Johnson & Johnson’s Opsynvi, the first and only once-daily combination pill of macitentan and tadalafil. Opsynvi, specifically for people with functional class II and III PAH, is expected to become available in mid-April.

On Tuesday, the FDA approved Merck’s Winrevair (sotatercept-csrk), the first PAH drug that works on a new biological pathway different from those targeted by 14 existing FDA-approved treatments.

The Pulmonary Hypertension Association is excited to see industry research that has led to better understanding of PAH and the development of new treatment options. Both treatments represent breakthroughs for the PH community. A once-daily pill offers increased convenience that can improve quality of life. And medication that works on a new pathway offers hope that PH can be reversed.

Opsynvi can be taken by people who currently take 10 mg a day of macitentan and 40 mg of tadalafil in separate tablets. Opsynvi is for people just starting treatment, as well as those who take endothelin receptor antagonists such as macitentan, phosphodiesterase 5 inhibitors such as tadalafil, or both. Existing clinical guidelines recommend initial combination therapy for idiopathic, heritable, drug-related or connective tissue disease-associated PAH.

Winrevair is a subcutaneous injection given every three weeks. The injection is taken in combination with other PAH treatments. People who took Winrevair in clinical trials showed significantly improved exercise capacity and slower disease progression.

Winrevair targets an imbalance in activin-growth differentiation factor and BMP pathways signaling. An activin signaling inhibitor, Winrevair works differently from previous PAH treatments. The other FDA-approved treatments target three pathways that don’t work properly in people with PH: the overactive endothelin pathway and underactive nitric oxide and prostacyclin pathways. Existing drugs are mostly vasodilators, which relax the blood vessels and help the blood flow better.

Winrevair acts on structural changes in the blood vessels. Those changes make the vessels thicker, harder and more rigid. Winrevair works to reduce and possibly reverse proliferation of cells in the vascular wall.

It’s been a long time since the FDA approved a PAH treatment. That was in 2015 when selexipag became available. More recently, the FDA approved inhaled Tyvaso for people whose PH is related to interstitial lung disease. Tyvaso helps relieve shortness of breath so people can exercise more easily.

Fortunately, several other new treatments are under development, including seralutinib, by Gossamer Bio and imatinib by Aerovate. Like Winrevair, those drugs modulate new pathways involved in long-term structural changes in the blood vessels. Seralutinib and imatinib inhibit certain enzymes that manage cell growth, but they work on different pathways from each other, as well as from Winrevair and existing PH treatments.

We welcome the development of new therapies for those with PAH. A PAH diagnosis is a life-changing experience for patients and families due to the chronic, progressive nature of the disease.

Despite the availability of 16 PAH treatments in the US, we know PH research has a long way to go. We still need medications for other forms of PH, and we need better accessibility to treatment in other parts of the world.

That’s why PHA supports PH research through grants and professional development opportunities for researchers, scientists and health care professionals. We encourage all PH professionals to attend PHA 2024 International PH Conference and Scientific Sessions, where they’ll hear from international PH experts, learn about research advancements and most importantly, meet the people affected by pulmonary hypertension.

We also encourage people with PH and caregivers to attend the Aug. 15-18 conference in Indianapolis. Some will be first-timers time, eager to learn about the disease and meet other patients and caregivers. Others will be looking to reconnect and learn more about treatment and care.

Whether you’re new to PH or a long-time thriver, we know how important it is to connect — not just with others affected by PH — but also with professionals who can answer your questions first-hand.

PHA 2024 registration opens Friday, March 29. PHA members receive discounted registration rates and may be eligible for scholarships and stipends to further lighten your financial burden, so make sure your PHA membership is up to date.

I hope to see you in Indianapolis in August. In the meantime, please let me know if you have questions or concerns. As always, I look forward to hearing from you.

With gratitude and respect,