The European Medicines Agency (EMA) recently accepted the Marketing Authorisation Application (MAA) filed by Actelion seeking approval for its pulmonary arterial hypertension (PAH) candidate, macitentan.
We note that the marketing application was submitted on the basis of data from a long-term, pivotal, and event-driven phase III outcome study (SERAPHIN: n=742). The study evaluated macitentan at two doses (10 mg and 3mg) in patients suffering from PAH. Results from the phase III study revealed that treatment with macitentan (10mg once daily) reduced the risk of morbidity and mortality event by 45% compared to placebo. The risk was reduced by 30% in PAH patients treated with the 3 mg dosage of the drug.
Data from the study also revealed that the risk of death or hospitalization due to PAH was 50% and 33% lower for patients in the 10 mg and 3 mg arms, respectively, compared to placebo. Patients in the study were treated for a period of three and a half years and the candidate was well tolerated among these patients. Actelion further reported that the number of serious adverse events was lower in patients treated with macitentan than placebo.
We note that macitentan is also under review in the US for the PAH indication. In October 2012, Actelion submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) seeking approval of macitentan for the indication. Actelion also intends to get macitentan approved for PAH in Switzerland and other major markets across the globe.
Actelion currently has three marketed products, Tracleer, Ventavis and Veletri, for the treatment of PAH. We remind investors that during the first nine months of 2012 the company reported a decline in sales in Tracleer and Ventavis from the year-ago period. Veletri, launched in 2010, however did very well during the period.
Actelion currently competes with players such as Gilead Sciences (GILD - Free Report) and United Therapeutics (UTHR - Free Report) in the PAH space. Actelion’s top line would be boosted further if macitentan is cleared in the US and the EU.
Switzerland-based Actelion carries a Zacks #1 Rank (Strong Buy) in the short run.