The FDA approved the oral dual endothelin-receptor blocker macitentan (Opsumit) for treatment of pulmonary arterial hypertension (PAH).
The drug is the first oral PAH agent approved with an indication to delay disease progression and to reduce hospitalization for the disease.
The pivotal SERAPHIN trial was the first in PAH to be powered for a hard clinical endpoint aside from change in functional class or 6-minute walk distance.
The 10-mg once daily dose reduced the composite risk of death, atrial septostomy, lung transplantation, initiation of intravenous or subcutaneous prostanoids, or worsening PAH by 45% compared with placebo in the trial.
The higher dose reduced the PAH hospitalization rate to 19% compared with 32% on placebo.
A lower, 3-mg dose reduced those risks more modestly and was not approved by the agency. Higher doses haven't been studied and are not recommended, according to the label.
Women will only have access to the drug through a Risk Evaluation and Mitigation Strategy (REMS) Program, due to risk of fetal harm.
Like other members of its class -- bosentan (Tracleer) and ambrisentan (Letairis) -- macitentan will carry a boxed warning against use in pregnant women.
Common adverse effects from macitentan include anemia, nasopharyngitis, sore throat, bronchitis, headache, flu, and urinary tract infection.
Macitentan is marketed by San Francisco-based Actelion Pharmaceuticals, which said in a release that the drug should be available on the market in November.
The announcement followed approval of another new PAH drug in a different class, riociguat (Adempas), earlier this month.