The Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), has given a positive opinion to Uptravi (selexipag) for the treatment of pulmonary arterial hypertension, according to Switzerland-based Actelion Pharmaceuticals.
A final decision by the European Commission is expected in April.
The drug has recently been approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult pulmonary arterial hypertension (PAH) patients.
PAH is a life-threatening disease characterized by abnormal constriction of the pulmonary artery, causing the heart to work faster and elevating blood pressure in arteries within the lungs, potentially leading to heart failure.
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 growth and proliferation of vascular smooth muscle cells.
Selexipag’s efficacy and safety were assessed and proved in a long-term Phase 3 clinical trial, the GRIPHON study. The study included 1,156 PAH patients, randomized to drug and placebo groups, and revealed 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 and morbility events, the primary endpoint of the trial, by 40 percent when compared to the control group. The most common reported adverse reactions of mild to moderate intensity were jaw pain, headache, diarrhea, and nausea, among others.
“We are delighted by today’s announcement of a positive CHMP opinion,” Dr. Jean-Paul Clozel, CEO of Actelion, said in a press release. “Coming hot on the heels of both the U.S. and Canadian approvals, we believe that Uptravi can significantly improve long-term outcomes for PAH patients. Once market authorization is granted by the EU Commission, Uptravi will open up the prostacyclin pathway to many more patients.”
The CHMP recommends approval of Uptravi by the European Community as a combination therapy for PAH in patients insufficiently controlled with endothelin receptor antagonist (ERA) and/or phosphodiesterase type 5 (PDE-5) inhibitors, or as a monotherapy in patients that are not eligible for these therapies. The treatment is recommended for adult patients with PAH with WHO functional class II-III.