EU regulators have issued a green light for Actelion's Uptravi, offering a new treatment option to patients with pulmonary arterial hypertension (PAH).
The drug has been licensed for the long-term treatment of PAH in adult patients with WHO functional class (FC) II-III, either as a combination therapy in patients insufficiently controlled with an endothelin receptor antagonist and/or a phosphodiesterase type 5 (PDE-5) inhibitor, or as monotherapy in those unable to take these therapies.
PAH is a progressive disease with high rates of morbidity and mortality affecting around 6,000-7,000 people in the UK. It is characterised by abnormally high blood pressure in the arteries between the heart and lungs, causing symptoms such as such as breathlessness, fatigue, weakness and angina.
While options are available to address these symptoms there is currently no treatment that slows progression or cures the disease, and fewer than 40% of patients live beyond five years of diagnosis.
Uptravi's (selexipag) approval came on the back of data from the Phase III GRIPHON study showing that the drug, a selective IP prostacyclin receptor agonist, delayed the time to a first morbidity or mortality event compared to placebo.
In the trial, exposure to the drug was up to 4.2 years with a median duration of exposure of 1.4 years; the risk of this primary composite end-point was reduced by 40 percent with Uptravi compared to placebo (a relative risk reduction of 16 percent).
"An innovative, oral treatment, supported by long-term outcome results will allow us to offer convenient triple combination therapy regimens that target these three established treatment pathways," said Professor Sean Gaine, consultant respiratory physician at Mater Misericordiae Hospital, Dublin. "With this approach we not only aim to delay disease progression but we are looking for long-term benefits for our patients."
"We are extremely proud to be able to offer the UK and Ireland pulmonary hypertension community the first orally active therapy that targets the prostacyclin pathway," said Robin Bhattacherjee, general manager at Actelion Pharmaceuticals UK.
"Bringing Uptravi to market, where currently the only licensed options are a 24-hour IV infusion or inhalation therapy, is a genuine advancement in the long-term treatment options for patients with PAH. We now hope to secure reimbursement, making Uptravi available to patients in the UK and Ireland."