FDA approves orphan drug selexipag for treatment of adults with PAH

FDA approves orphan drug selexipag for treatment of adults with PAH

The orphan drug selexipag (Uptravi, Actelion) has been approved by the US Food and Drug Administration (FDA) for treatment of adults suffering from pulmonary arterial hypertension (PAH). PAH is a chronic and progressive rare lung disease that can cause premature death or require transplantation.

An oral IP prostacyclin receptor agonist, Selexipag, relaxes muscles to widen blood vessels and reduces the elevated pressure in the vessels that supply blood to the lungs.

Selexipag’s safety and efficacy has been shown in a clinical trial that included 1156 adults suffering from PAH who underwent treatment for a median of 1.4 years. The FDA said that the drug succeeded in decreasing hospitalization for PAH and the disease progression risk compared with placebo.

In a news release, the company said that selexipag’s benefit was consistent across pre-specified patient subgroups like functional class, disease etiology and baseline PAH therapy that also included patients already getting combination therapy with a PDE-5 inhibitor and an endothelin receptor antagonist.

The trial saw many adverse effects, and some of the common ones included diarrhea, jaw pain, headache, nausea, vomiting, extremity pain, myalgia, and flushing.

In a statement, Ellis Unger, MD, from the FDA's Center for Drug Evaluation and Research, said that selexipag offers an extra treatment option for patients suffering from PAH.

Jean-Paul Clozel, Actelion Chief Executive Officer said, “Selexipag will significantly expand the options to delay disease progression after initiation of therapy with a baseline treatment like Opsumit (macitentan), and well ahead of Veletri (epoprostenol) for the late disease stage”.