Veletri (epoprostenol) is a vasodilator — a medication that opens, or dilates, blood vessels — marketed by Actelion Pharmaceuticals. It is approved in the U.S. for the treatment of pulmonary arterial hypertension (PAH), including PAH associated with connective tissue diseases, such as scleroderma.

PAH is caused by the narrowing and stiffening of blood vessels in the lungs, leading to increased resistance and high blood pressure.

Veletri can also improve Raynaud’s phenomenon, a disorder in which the fingers and toes often feel numb and cold, and their blood vessels become very narrow. Many scleroderma patients develop Raynaud’s phenomenon as a secondary disease.

How Veletri works

The active ingredient of Veletri, epoprostenol, is a synthetic version of a naturally occurring hormone called prostacyclin. This hormone is a potent vasodilator, and is normally produced by cells lining the pulmonary arteries.

It binds to prostacyclin receptors found on platelets — blood cells that help form clots to stop bleeding — as well as the cells lining the pulmonary arteries themselves. This binding activates signaling cascades within the cells, which blocks platelet aggregation, and to the dilation of the pulmonary arteries. Through these two mechanisms, prostacyclin is thought to decrease the pressure in pulmonary arteries.

Veletri in clinical trials

Intravenous epoprostenol (Veletri) has been used to treat PAH for many years. Only a few studies assessed the effectiveness of epoprostenol specifically in patients with scleroderma-associated PAH.

In an open-label, randomized trial, 111 PAH patients received either epoprostenol in combination with a conventional therapy or conventional therapy alone.

After 12 weeks of treatment, the exercise capacity of patients receiving the combination treatment improved significantly compared to those who received conventional therapy alone.

The mean pulmonary artery pressure and the mean pulmonary vascular resistance (the force that opposes the blood flow through a vessel) decreased in patients receiving epoprostenol and increased in those who did not.

In an open-label three-year extension study, 102 scleroderma-associated PAH patients received epoprostenol. The study reported an increased survival rate compared to historical data where patients received conventional therapy.

A Phase 4, open-label trial (NCT01105091) in 30 PAH patients compared the safety and tolerability of Veletri to Flolan, another epoprostenol formulation, which was previously used to treat PAH.

Most adverse events were classified as mild, and both groups of patients had a comparable adverse event profile. Veletri has a higher stability at room temperature compared to Flolan.

Additional information

Epoprostenol is administered with the help of an intravenous catheter, which may cause serious complications such as obstruction, mechanical malfunction, and infection.

The most common side effects of Veletri include headaches, flushing, nausea, low blood pressure, slow or fast heartbeat, abdominal pain, shortness of breath, anxiety, and dizziness.

PAH patients with an underlying connective tissue disease taking Veletri are more prone to develop a lack of appetite, nausea, diarrhea, and jaw, neck, and joint pain, as well as headache, low blood pressure, and skin ulcers, compared to patients receiving conventional therapy.

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