The Pulmonary Hypertension Association of Canada (PHA Canada) is applauding the province of Quebec’s decision to publicly fund the drug Opsumit as a new treatment option for pulmonary arterial hypertension (PAH).
PAH is high blood pressure that occurs in the arteries that connect the heart to the lungs, and can result in limitations on exercise ability and shortness of breath. PAH is life-threatening and strikes apparently at random, regardless of age, sex, social or ethnic background. People affected with PAH suffer from continuous high blood pressure in the lungs, causing the right side of the heart to work harder than normal, which results in enlargement of the heart that can lead to heart failure. PAH is difficult to diagnose because it shares symptoms with many other conditions, and consequently many patients are misdiagnosed. There is currently no cure. It is estimated that up to 10,000 Canadians may be affected by PAH.
Some PAH symptoms are shortness of breath; bluish hands, feet and lips; swelling of hands and feet; light-headedness and dizziness; chest pain; exhaustion and fainting.
Opsumit (macitentan) is a novel dual endothelin receptor antagonist (ERA), which is taken orally once daily. It belongs to a class of drugs called endothelin receptor blockers, which act to relax the pulmonary arteries, decreasing blood pressure in the lungs. Opsumit resulted from a tailored drug discovery process in the laboratories of San Francisco-based Actelion Pharmaceuticals, a biopharmaceutical company focused on the discovery, development, and commercialization of innovative drugs for diseases with significant unmet medical needs.
On October 1, the Quebec Institut national d’excellence en santé et services sociaux (INESSS) recommended that Opsumit (macitentan) be approved for treatment of (PAH), and it will be added to the list of drugs covered by the prescription drug insurance plan administered by the Régie de l’assurance maladie du Québec (RAMQ).
“This news marks an important milestone for patients, their caregivers and their physicians in Quebec who now have access to a new treatment option that can offer a better quality of life to some patients,” says Denis Cormier, President of the Fondation Hypertension Artérielle Pulmonaire – Québec (HTAPQ). “Clinical studies on Opsumit provide us with long-term data, which is rare in PAH research, and brings much hope to patients and their families.”
The U.S. Food and Drug Administration approved Opsumit to treat adults with PAH in 2013, based on Opsumit’s safety and effectiveness established in a randomized, placebo-controlled study of PAH patients called SERAPHIN — the first long-term study to include a clearly defined, clinically-important morbidity/mortality primary endpoint. In the study, 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.
Similar to other members of its drug class, Opsumit carries a Boxed Warning, alerting patients and health care 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.
Common side effects observed in those treated with Opsumit include low red blood cell count (anemia), common cold-like symptoms (nasopharyngitis), sore throat, bronchitis, headache, flu and urinary tract infection.
Opsumit was approved in November 2013 by Health Canada for long-term treatment of pulmonary arterial hypertension to reduce morbidity, but Quebec is the first Canadian province to recommend Opsumit for public reimbursement. PAH advocates’ hope is that all provinces and territories will follow Quebec’s lead and make Opsumit available to all Canadians who can benefit from it.
Macitentan is currently being studied in the Phase III study MAESTRO (MAcitentan in Eisenmenger Syndrome To RestOre exercise capacity) — multi-center, double-blind, randomized, placebo-controlled, parallel-group, Phase 3 study to evaluate the effects of macitentan on exercise capacity in subjects with Eisenmenger Syndrome. This study is ongoing with approximately 220 subjects to be randomized in a 1:1 ratio into 2 treatment groups (macitentan 10mg or placebo) over a 16 week treatment period.
“The PAH community across the country applauds the Quebec government for its leadership in providing funding for this important new treatment option, which puts an additional tool in physician’s hands to help improve the quality of life of those suffering from this deadly and aggressive lung disease,” says Angie Knott, National Manager, Pulmonary Hypertension Association of Canada’. “We urge all provincial and territorial governments to give patients across Canada who can benefit from Opsumit the same breath of hope. Every patient living with this disease is different, so the more treatment options that physicians have at their disposal to treat PAH, the more lives can be saved.”
The Pulmonary Hypertension Association of Canada (PHA Canada) is a national, registered charity working to end the invisibility and isolation often experienced by persons living with this life-changing disease. To learn more about PHA Canada and pulmonary hypertension, visit:
Pulmonary Hypertension Association of Canada
U.S. Food and Drug Administration