Opsumit approved to treat pulmonary arterial hypertension

FDA approved macitentan (Opsumit, Actelion Pharmaceuticals) to treat adults with pulmonary arterial hypertension (PAH), a chronic, progressive and debilitating disease that can lead to death or lung transplantation.

“The FDA approval of Opsumit for PAH serves as another landmark drug approval in the agency's effort to provide safe and effective drug options to the public, and also help to improve the quality of life of those who live with and suffer from the limitation of PAH,” said Formulary advisor Abimbola Farinde, PharmD, MS, clinical staff pharmacist at Clear Lake Regional Medical Center, Webster, Texas.

Opsumit belongs to a class of drugs called endothelin receptor blockers, 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 2 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.

Like 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. Female patients can receive the drug only through the Opsumit Risk Evaluation and Mitigation Strategy (REMS) Program. This restricted-distribution program requires prescribers to be certified by enrolling in the program; all female patients to be enrolled in the program and comply with applicable pregnancy testing and contraception requirements before initiating treatment; and pharmacies to be certified and to dispense Opsumit only to patients who are authorized to receive it.

Low red blood cell count (anemia), common cold-like symptoms (nasopharyngitis), sore throat, bronchitis, headache, flu, and urinary tract infection are common side effects observed in those treated with Opsumit.

According to the American Lung Association, PAH is a disease in which the pressure in a patient's pulmonary arteries becomes dangerously high. Pulmonary arteries carry blood that has returned from the body to the lungs, where the blood receives a fresh supply of oxygen. That high blood pressure puts a strain on the heart.  PAH is 1 of 5 types of pulmonary hypertension. PAH worsens over time and is life-threatening. It affects 1 in 100,000 to 1 in 1 million people.