The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for a variation to expand the current therapeutic indication for GlaxoSmithKline's Volibris (ambrisentan) to include its use in combination treatment for patients with pulmonary arterial hypertension (PAH).
The CHMP opinion was based on data from the Phase IIIb/IV Ambition trial, which is the first and only outcomes study to compare the efficacy and safety of investigational first-line combination therapy of ambrisentan and tadalafil to first-line monotherapy of either treatment alone in treatment-naïve patients with PAH.
Results from this trial showed that the combination of ambrisentan and tadalafil reduced the risk of clinical failure by 50% in treatment-naïve PAH patients compared to pooled ambrisentan and tadalafil monotherapy.
The Phase IIIb/IV Ambition trial was co-sponsored by GSK and Gilead, with support provided by Eli Lilly.
"If approved, this could help improve clinical outcomes for patients across Europe suffering from this rare condition.GSK Rare Diseases clinical investigation lead PAH Jonathan Langley said: "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.
"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 company noted that the positive opinion is a formal recommendation by the CHMP to expand the existing marketing authorisation for ambrisentan, a selective endothelin type-A receptor antagonist, to include combination treatment.
The European Commission will review the CHMP's positive opinion and a final decision is expected to be made by December this year.
Further regulatory submissions to include the Ambition data in the ambrisentan label are ongoing and planned in other countries.
Ambrisentan is approved in the EU and other countries as a once-daily treatment for PAH, (WHO Group 1) in patients with WHO/NYHA functional class II and III symptoms.
In territories outside of the US, GSK commercialises ambrisentan under the tradename Volibris and Gilead commercialises the product under the tradename Letairis in the US.
The product has also been granted orphan drug status for the treatment of PAH in Australia, Europe, Japan, Korea and US.
