On Dec. 21, the U.S. Food and Drug Administration approved Uptravi (Selexipag) tablets to treat adults with pulmonary arterial hypertension (PAH), a chronic, progressive, debilitating, and rare lung disease that can lead to death or the need for transplant.
PAH is high blood pressure that takes place in the arteries which connect the heart to the lungs. It causes the right side of the heart to work harder than normal, which can lead to limitations on exercise ability and shortness of breath, among other more serious complications. Uptravi belongs to a class of drugs called oral IP prostacyclin receptor agonists.
The drug acts by relaxing muscles in the walls of blood vessels to dilate (open) blood vessels and decrease the elevated pressure in the vessels supplying blood to the lungs. The drug is a first-in-class selective agonist of the prostacyclin IP receptor, one of the five major types of prostanoid receptors, which is involved in vasodilation and inhibition of vascular smooth muscle cell proliferation.
“Uptravi offers an additional treatment option for patients with pulmonary arterial hypertension,” said Ellis Unger, M.D., director of the Office of Drug Evaluation I in the FDA’s Center for Drug Evaluation and Research. “The FDA supports continued efforts to provide new treatment options for rare diseases.”
The drug was tested and evaluated in clinical trials involving 1,156 adults with pulmonary arterial hypertension. Trial participants who took the drug did so for an average of about a year and a half. The drug was found to limit worsening of the disease and reduce the need for hospitalization. Uptravi’s most common side effects included headache, diarrhea, jaw pain, nausea, myalgia, vomiting, pain in the arms and legs, and flushing.
The drug is marketed by San Francisco-based Actelion Pharmaceuticals. The findings were consistent across key subgroups including background therapies, WHO functional class, and PAH etiology in the trial (58 percent idiopathic and heritable, 29 percent associated with connective tissue disease, 10 percent associated with congenital heart disease with repaired shunts).
“The label for Uptravi recognizes the improvement in long-term outcomes, including reducing the risk of hospitalization for PAH regardless of whether patients received background therapy including an ERA [endothelin receptor antagonist], a PDE [phosphodiesterase]-5 inhibitor, or — for the first time ever in PAH — on top of a combination of both, an ERA and a PDE-5 inhibitor,” Jean-Paul Clozel, MD, chief executive officer of Actelion, said in a press release.
Uptravi’s safety and efficacy were established in a long-term clinical trial of 1,156 participants with PAH. Uptravi was shown to be effective in reducing hospitalization for PAH and reducing the risks of disease progression compared to placebo. Participants were exposed to Uptravi in this trial for a median duration of 1.4 years.
The FDA, an agency within the U.S. Department of Health and Human Services, promotes and protects the public health by, among other things, assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines, and other biological products for human use and medical devices. The drug will be available to U.S. patients in early January 2016.
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