A rare heart disease called Pulmonary Arterial Hypertension (PAH) may now be treated orally using a drug called Selexipag, revealed a new study. The study will be published in the Dec. 24 issue of the New England Journal of Medicine.
Data from the largest study ever of pulmonary hypertension shows the oral medication Selexipag led to a 40 percent reduction in hospitalizations and worsening symptoms among patients with pulmonary hypertension.
Selexipag targets a well-known disease pathway that opens blood vessels to the lungs and improves heart function and is easier to use than PH treatments delivered with infusions or injections. It was approved by the U.S. Food and Drug Administration this week, setting the course for it to be available for patients in January.
"But because of the cumbersome nature of treatment, patients would often wait until late stages to begin therapy. Having an oral medication to attack the disease pathway will be a major advancement because less ill patients will be willing to begin this therapy," McLaughlin said.
Selexipag demonstrated effectiveness while its most frequent adverse events were headache, diarrhea, nausea, muscle pain and joint pain. The side effects were considered consistent with prostacyclin therapy.
Pulmonary hypertension, which is high blood pressure in the loop of blood vessels connecting the heart and lungs, can make everyday activities exhausting. It often leads to life-threatening heart failure as the heart works harder to pump blood to the lungs.
The greatest number of cases is reported in women between ages 21 and 40 who most often experience fatigue and shortness of breath.
While a rare disease, research of causes and treatment of pulmonary hypertension is growing. The GRIPHON study is a standout because of its size and scope, enrolling 1,156 patients with pulmonary hypertension from 181 centers from 39 countries in North and South America, Europe, Asia-Pacific and Africa.
Collaborators from the U.S. and Europe led the GRIPON study, which was sponsored by Actelion Pharmaceuticals.