Actelion has announced that its orally active pulmonary arterial hypertension (PAH) drug, Uptravi (selexipag), has been approved for the treatment of PAH patients by both the Therapeutic Goods Administration of Australia and the New Zealand Medicines and Medical Devices Safety Authority.
The drug was recently approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult PAH patients, and the Committee for Medicinal Products for Human Use (CHMP), the scientific arm of the European Medicines Agency (EMA), has given a positive opinion of the medicine. This opinion is currently under review, and a final regulatory decision by the EU commission is expected in April.
Selexipag is a highly selective oral prostacyclin receptor (IP) agonist originally discovered and synthesized by Nippon Shinyaku. Activation of the IP receptor induces vasodilation, which decreases blood pressure and inhibits the growth and proliferation of vascular smooth muscle cells. It is indicated for the treatment idiopathic PAH, heritable PAH, PAH associated with connective tissue disease, PAH associated with congenital heart disease with repaired shunts, and PAH associated with drugs and toxins, in patients with WHO functional class II, III or IV symptoms.
The Phase 3 placebo-controlled GRIPHON study, which assessed selexipag’s efficacy and safety, contributed significantly to the decision. The study included 1,156 PAH patients and reported long-term benefits of the drug at a tolerable maintenance dose. At the end of treatment, therapy with selexipag significantly decreased the risk of morbidity or mortality events, the primary endpoint of the trial, by 40 percent when compared to the control group. The most common adverse events, which included jaw pain, headache, diarrhea, and nausea, were mild to moderate in intensity.
“The approval of Uptravi represents a major step forward in disease management for the PAH communities in both Australia and New Zealand. Until now the options for treatments targeting the prostacyclin pathway have been limited, and were burdensome for the patient,” Simon Eade, head of Actelion Asia Pacific Region, said in a press release. “Uptravi offers patients an oral treatment that targets the prostacyclin pathway, opening the way for oral combination therapies with proven long-term outcome benefits. We will now work to secure reimbursement and make Uptravi available to patients.”