Medication Monitor



Generic Name (Trade Name—Company)
Notes
December 22, 2015

Selexipag

(Uptravi—Actelion)
New orphan drug treats pulmonary arterial hypertension

FDA approved selexipag tablets to treat adults with pulmonary arterial hypertension (PAH), a chronic, progressive, and debilitating rare lung disease that can lead to death or the need for transplantation.

Selexipag belongs to a class of drugs called oral IP prostacyclin receptor agonists. The drug acts by relaxing muscles in the walls of blood vessels to dilate blood vessels and decrease the elevated pressure in the vessels supplying blood to the lungs.

Safety and efficacyof the drug were established in a long-term clinical trial of 1,156 participants with PAH. Selexipag was shown to be effective in reducing hospitalization for PAH and reducing the risks of disease progression compared with placebo. Participants were exposed to selexipag in this trial for a median duration of 1.4 years.  

Common adverse effects observed in those treated with selexipag in the trial include headache, diarrhea, jaw pain, nausea, muscle pain, vomiting, pain in an extremity, and flushing.

Selexipag was granted orphan drug designation, which provides incentives such as tax credits, user fee waivers, and eligibility for exclusivity to assist and encourage the development of drugs for rare diseases.