Macitentan, a recent addition to the drugs that treat pulmonary arterial hypertension (PAH), improves and stabilizes quality of life for patients with the condition, according to an industry-funded study.
Macitentan (Opsumit) remains tremendously expensive, costing as much as $100,000 per year in the United States, and the study provides little in the way of direct comparison to other drugs in its class. Still, the drug’s effects on quality of life are dramatic, said study lead author Sanjay Mehta, MD, FRCPC, FCCP, professor of medicine at the University of Western Ontario and director of the Southwest Ontario Pulmonary Hypertension Clinic at the London Health Sciences Center in London, Ont.
Researchers found that those who took the 10-mg dose, versus placebo, reported significant improvement in seven of eight quality-of-life domains, and in physical and mental components scores, as measured by the 36-item Short Form Health Survey (SF-36). In addition, the study linked 10-mg doses, versus placebo, to a lower risk of a decline of three points or more in the physical component score (hazard ratio [HR], 0.60; 95% CI, 0.47-0.76; P less than .0001] and the mental component scores (HR, 0.76; 95% CI, 0.61-0.95; P = .0173) until end of treatment.
“The drug has shown stability in patients’ quality of life over 6 months and 12 months,” Dr. Mehta said in an interview. “I can’t cure anybody, and they’ll get worse at some point, but I can improve them. They physically feel better, they’re less short of breath with less body pain, and they feel better psychologically.”
Macitentan, an endothelin receptor antagonist, received Food and Drug Administration approval in 2013 following a study that year (N Engl J Med. 2013 Aug 29;369[9]:809-18) that linked 10-mg doses to a significantly lower risk of death and various complications, compared with placebo and the 3-mg dose. The new study (Chest. 2017 Jan;151[1]:106-18), is an analysis of data from the 2013 study.
The PAH patients were randomly assigned to one of three groups: macitentan 10 mg once daily (234), macitentan 3 mg (237), and placebo (239). The study examined responses from 710 patients (76.9% were female, 55.2% were white, mean age was 45.5) to the SF-36 at baseline, 6 months, 12 months, and end of treatment.
Dr. Mehta noted that macitentan has not been clinically compared to the other drugs. The study, however, notes that it is the first PAH treatment to show improvement in seven of eight domains in the quality-of-life survey.
The new study was funded by Actelion Pharmaceuticals, maker of macitentan. Dr. Mehta has received consulting and speaking fees and institutional support for clinical trials from Actelion, among other drug companies. The other authors report various disclosures, including relationships with Actelion.