GSK’s Application to Expand PAH Drug Volibris’ (ambrisentan) Therapeutic Indication as Combined Treatment Receives Positive CHMP Opinion

GlaxoSmithKline (GSK) recently announced that the European Medicine Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has released a positive recommendation for its application to expand the existing therapeutic indication for pulmonary arterial hypertension (PAH) combination treatment Volibris® (ambrisentan) — a selective endothelin type-A receptor antagonist. The therapy is currently approved in the EU and other territories as a once-a-day treatment for PAH patients with WHO/NYHA functional class II and III symptoms.

“Today’s decision represents an important milestone for the PAH community, supporting the potential for initial combination treatment in this highly debilitating and limiting disease,” said Jonathan Langley, Clinical Investigation Lead PAH, GSK Rare Diseases. “If approved, this could help improve clinical outcomes for patients across Europe suffering from this rare condition. We look forward to receiving a final decision from the European Commission in the coming months.”

The CHMP’s opinion drew from the positive results of Phase 3b/4 AMBITION study, which was the first and only comparative outcome study that evaluated the efficacy and safety profile of ambrisentan/tadalafil against monotherapy with either of the two agents in patients who have never received treatment for PAH. The study, titled, “Initial Use of Ambrisentan plus Tadalafil in Pulmonary Arterial Hypertension” was published in the New England Journal of Medicine and successfully demonstrated the combination’s significantly reduced rate of clinical failure (by 50 percent) in treatment-naive patients, compared to those that received monotherapy.

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This CHMP recommendation contributes to the pending decision of the European Commission on expanding the drug’s indication, which is expected by December 2015. Other regulatory submissions based on the data from AMBITION will soon be filed, or are already under review.

Pulmonary arterial hypertension is a common consequence of various chronic obstructive pulmonary diseases, characterized by an increase in blood pressure in pulmonary arteries due capillary obstruction, narrowing and degradation. If not treated, the disease can lead to death due to cardiorespiratory disorders and heart failure in a relatively short time frame.