Three new drugs for pulmonary hypertension have been approved by the US and/or EU regulatory authorities in the course of the last year:
Macitentan (Opsumit), a new drug to treat adults with pulmonary arterial hypertension (PAH) was approved by the U.S. Food and Drug Administration (FDA) on October 18, 2013 and by the European Medicines’ Agency (EMA) on December 20, 2013.
Opsumit belongs to a class of drugs called “endothelin receptor antagonists”, which act to relax the pulmonary arteries, decreasing blood pressure in the lungs. Opsumit’s safety and effectiveness were established in a long-term clinical trial where 742 participants were randomly assigned to take Opsumit or placebo. The average treatment duration was about two years. In the study, Opsumit was effective in delaying disease progression, a finding that included a decline in exercise ability, worsening symptoms of PAH or need for additional PAH medication. Opsumit is marketed by San Francisco-based Actelion Pharmaceuticals US, Inc.
Riociguat (Adempas), a new drug to treat adults with pulmonary arterial hypertension (PAH) and chronic thromboemblic pulmonary hypertension (CTEPH) was approved by the the U.S. Food and Drug Administration (FDA) on October 8, 2013 and by the European Medicines Agency (EMA) on March 27, 2014.
Adempas belongs to a class of drugs called soluble guanylate cyclase stimulators that help arteries relax to increase blood flow and decrease blood pressure. It is intended for patients with chronic thromboembolic pulmonary hypertension (CTEPH) after surgery or patients who cannot undergo surgery, to improve their ability to exercise. Adempas is also indicated for patients with pulmonary arterial hypertension (PAH) of unknown causes, inherited or associated with connective tissue diseases, to improve their ability to exercise and to delay clinical worsening of their condition.The clinical trial evaluating the safety and effectiveness of Adempas to treat PAH included 443 participants randomly assigned to take Adempas 1.5 mg or 2.5 mg, or placebo, three times daily. After 12 weeks of treatment, the 6-minute walk distance in patients treated with Adempas improved by an average of 36 meters (about 118 feet) more than in patients treated with placebo.Adempas is marketed by Bayer HealthCare Pharmaceuticals Inc., based in Wayne, N.J.
On December 20, 2013, the U.S. Food and Drug Administration (FDA) approved Orenitram (treprostinil) extended-release tablets for the treatment of pulmonary arterial hypertension (PAH) in WHO Group I patients to improve exercise capacity. The primary study that established efficacy (FREEDOM-M) included predominately patients in WHO functional class II-III and etiologies of idiopathic or heritable PAH (75%) or PAH associated with connective tissue disease (19%).Orenitram is a trademark of United Therapeutics corporation, Silver Spring, Md,USA.