Pulmonary arterial hypertension (PAH), a chronic condition marked by high blood pressure in the pulmonary arteries that connect the heart and lungs, only affects about 15 to 50 individuals per 1 million. Despite the low prevalence of the disease, however, it is potentially fatal for those suffering from the condition.
Symptoms of PAH range from fatigue and breathlessness to more severe complications that could lead to early death such as heart failure. Medications that can reduce the pressure on the heart and treat the symptoms, however, are available. With the promising results of a phase III study of a new experimental drug developed by Swiss biotech company Actelion Ltd. for pulmonary arterial hypertension, PAH sufferers may soon have another option for managing their condition.
Actelion announced on Monday that selexipag, its new experimental drug for the treatment of pulmonary arterial hypertension, had positive results in a double-blind and placebo-controlled phase III trial. Selexipag is also Actelion's third drug for the treatment of PAH.
Unlike other prostacyclin therapies that need to be inhaled or administered intravenously, selexipag, which was originally discovered by Japan-based pharmaceutical company Nippon Shinyaku, is taken orally. Selexipag is also Actelion's third drug for the treatment of PAH.
For the phase III GRIPHON study, the largest randomized and controlled study involving PAH patients with participants coming from 39 counties in Europe, Asia-Pacific, Africa, North America and Latin America, 1,156 individuals with PAH were given either placebos or selexipag two times daily.
After 4.3 years, investigators found that patients who took selexipag had reduced morbidity/mortality risks by 39 percent compared with patients who given placebos. The results were also found to be consistent regardless of age, gender and background therapy.
"The GRIPHON study results hold the promise that selexipag might open up the prostacyclin pathway to different groups of patients given the consistent efficacy findings across key subgroups evaluated in this long-term outcome study," said University of Michigan's Pulmonary Hypertension Program director Vallerie McLaughlin.
Actelion, however, said that 14 percent of the participants who took selexipag and seven percent of those in the placebo group discontinued treatment because of adverse events. The drug had adverse effects similar to those associated with other prostacyclin therapies including diarrhea, headache, nausea, jaw pain, vomiting, pain in the extremities, myalgia, or muscle pain, cold and flushing.
Actelion said that detailed results of the study will be published in a peer reviewed publication.