Ventavis (iloprost) is an approved therapy, developed by Schering AG (now part of Bayer), to improve exercise capacity in people with pulmonary arterial hypertension (PAH) and to potentially delay or reduce disease worsening.

It is also being used to treat patients with PAH, Raynaud’s phenomenon, and digital ulcers associated with systemic scleroderma.

How iloprost works

Iloprost, the active component of Ventavis, is a synthetic version of prostacyclin, a natural hormone produced by cells in the walls of blood vessels. Prostacyclin triggers signaling pathways that cause blood vessels to relax and widen.

Ventavis is an inhaled formulation of iloprost that is administered with a nebulizer. Because Ventavis is delivered directly to the lungs, it causes the dilation of narrowed blood vessels (arteries) in the lungs, relaxing and widening pulmonary arteries and reducing blood pressure to the heart. This leads to better oxygen transport throughout the body, improving patients’ ability to exercise.

Iloprost in clinical trials for systemic scleroderma

The efficacy of different doses of long-term iloprost, given intravenously, in treating Raynaud’s, ulcer healing, and skin thickening in patients with systemic sclerosis was evaluated in a Phase 2 clinical trial (NCT00622687).

During the trial, which ended in 2007, 50 patients with systemic scleroderma received a maximum dose of up to 2 ng/kg body weight per minute or a low-dose of 0.5 ng/kg body weight per minute iloprost administered for six hours daily over 21 days. Researchers assessed the effects of treatment on lung function by measuring forced vital capacity (the amount of air exhaled after taking the deepest breath possible) and predicted diffusion capacity (the extent of oxygen that passes from the lungs into the blood). Researchers also evaluated the effect of the medicine on Raynaud’s phenomenon, ulcer healing, skin thickness, and esophageal function. 

Study results, published in The Journal of Rheumatology, showed that the forced vital capacity and diffusion capacity of the lung were stable in 87 percent and 74 percent of treated patients, respectively.  In addition, both therapy regimens yielded a 70 percent reduction in digital ulcers, a 40 percent reduction in the frequency, and a 30 percent reduction in the duration of Raynaud’s phenomenon. Mild side effects were common in both groups but did not lead to discontinuation of therapy.   

A more recent study of iloprost in systemic sclerosis patients, conducted in Italy, found that long-term treatment (iloprost given intravenously about one week of each month for seven years) may stabilize and improve heart and lung function. Findings also showed that treatment improved the modified Rodnan skin score (a measure of sclerosis in the skin), and led to the healing of digital ulcers and prevented new ones from forming. These results were published online in October 2016 in the scientific journal Rheumatology International journal.

The effectiveness of long-term iloprost in treating digital ulcers in systemic scleroderma patients was reported in a study published in the Clinical and Experimental Rheumatology journal. Data showed that of the 31 of 50 patients with digital ulcers at the beginning of the trial, ulcers resolved in 22 (71 percent) patients during follow-up, suggesting that iloprost can be effective with long-term use to heal and prevent digital ulcers in systemic scleroderma patients. 

Other details

The most common side effects of Ventavis use include back pain, dizziness, flushing, headache, cough, jaw tightness, muscle cramps, nausea, sleeping disorders, and weakness.

Ventavis is marketed by Bayer in Europe, and by Actelion in the U.S.

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