Actelion Ltd. (ALIOF) recently announced the receipt of Japan's Ministry of Health, Labor and Welfare approval for epoprostenol "ACT" for the treatment of pulmonary arterial hypertension (PAH).
The product is marketed in the US as Veletri. In 2012, it gained marketing approval in Switzerland as Veletri and in Canada as Caripul. The candidate is currently under regulatory review in the EU.
We are encouraged by Japan’s approval of this candidate. We note that Actelion is progressing with its pipeline. Important pipeline candidates include ponesimod, Opsumit and cadazolid.
Ponesimod is being evaluated for plaque psoriasis. In Dec 2012, the company announced positive results from a phase II study for this indication. Results from the study showed that both doses of ponesimod (20 mg and 40 mg) achieved statistical significance. Actelion now plans to initiate a phase III study on ponesimod for the psoriasis indication.
In Apr 2012, Actelion reported positive results on Opsumit (macitentan) for the treatment of PAH. Actelion filed for US Food and Drug Administration (FDA) and European Medicines Agency (EMA) approval of Opsumit in the fourth quarter of 2012.
Cadazolid, for the treatment of clostridium difficile associated diarrhea (CDAD), is expected to move into phase III study in the current year. The decision to advance the candidate into late-stage development was based on an exploratory phase II dose-finding study.
Results showed that the effect of all doses of cadazolid were either numerically similar or better than Vancocin on primary endpoints, which included CDAD cure rates as well as sustained cure rates.