The US Food and Drug Administration (FDA) has approved macitentan (Opsumit, Actelion) to treat pulmonary arterial hypertension (PAH) in adults. The approval is the second this month for the progressive, debilitating disease.
The agency approved riociguat (Adempas, Bayer), for the treatment of PAH and the treatment of chronic thromboembolic pulmonary hypertension (CTEPH), on October 8.
Support for macitentan's approval comes from the 742-patientSERAPHIN trial, as reported by heartwire . In SERAPHIN, published in August 2013 in the New England Journal of Medicine, macitentan given at 10 mg/day over two years led to a 45% reduction (p<0.001) in a clinical primary end point that included death, initiation of IV or subcutaneous prostanoids, or worsening of PAH. The benefit, authors noted at the time, was driven primarily by reductions in PAH worsening.
In approving the agent today, the agency highlighted some caveats. "Similar to other members of its drug class, Opsumit carries a boxed warning alerting patients and healthcare professionals that the drug should not be used in pregnant women, because it can harm the developing fetus," a press release notes. "Female patients can receive the drug only through the Opsumit risk evaluation and mitigation strategy (REMS) program."
According to the REMS program, prescribers and pharmacies dispensing the drug need to be certified in the program, as do female patients taking the drug. Specifically, female patients need to comply with pregnancy testing and contraception requirements before initiating treatment.
The most common side effects seen with macitentan are anemia, common coldlike symptoms (nasopharyngitis), sore throat, bronchitis, headache, influenza, and urinary-tract infection.
Food and Drug Administration. FDA approves Opsumit to treat pulmonary arterial hypertension [press release]. October 18, 2013. Available here.