FDA accepts filing of Uptravi (selexipag) for treatment of PAH- Actelion

Actelion Ltd announced the formal acceptance of the New Drug Application (NDA) for selexipag (Uptravi) by the FDA. The NDA dossier for selexipag in the treatment of pulmonary arterial hypertension (PAH) was submitted to the FDA on 22 December 2014. Actelion expects results from the review process 12 months from the date of NDA submission. The FDA application for selexipag, the first selective oral prostacyclin IP receptor agonist, is based on the findings of the positive pivotal Phase III GRIPHON study in 1,156 patients with pulmonary arterial hypertension (PAH).

The GRIPHON study demonstrated that selexipag decreased the risk of a morbidity/mortality event versus placebo by 40% (p<0.0001). Efficacy observed was consistent across the key subgroups: age, gender, WHO Functional Class, PAH etiology and background PAH therapy. Patients were treated for up to 4.2 years. The tolerability profile of selexipag in GRIPHON was consistent with prostacyclin therapies. Adverse reactions occurring more frequently (>5%) on selexipag compared to placebo are headache, diarrhea, jaw pain, nausea, myalgia, vomiting, pain in extremity, and flushing.

Comment: Selexipag is the first oral long acting prostacyclin agonist. Actelion already has in place Opsumit (macitentan) which is a follow up to successful Tracleer (bosentan) due to be off patent in 2015. Its a competitive market in PAH that includes Letairis (ambrisentan) from Gilead and newly approved Adempas (riociguat) from Bayer Healthcare.