FDA approves drug for rare lung disease

December 23, 2015

The FDA recently granted orphan drug approval to Uptravi (selexipag) tablets to treat adults with pulmonary arterial hypertension (PAH), a chronic, progressive, and debilitating rare lung disease that can lead to death or the need for transplantation.

FDA granted orphan drug approval to Uptravi (selexipag) tablets to treat adults with pulmonary arterial hypertension (PAH), a chronic, progressive, and debilitating rare lung disease that can lead to death or the need for transplantation.

“Uptravi offers an additional treatment option for patients with pulmonary arterial hypertension,” said Ellis Unger, MD, director of the Office of Drug Evaluation-I in FDA’s Center for Drug Evaluation and Research.

Manufactured by Actelion Pharmaceuticals US, 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 blood vessels and decrease the elevated pressure in the vessels supplying blood to the lungs.

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Uptravi joins Opsumit, a baseline therapy for PAH, and late-stage treatment Veletri in Actelion's portfolio. “Actelion now has an unparalleled portfolio of treatments across the continuum of care in PAH that offer a combination of long term-efficacy, safety and convenience," said CEO Jean-Paul Clozel in a statement.

PAH is high blood pressure that occurs in the arteries that 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’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.

Common side effects observed in those treated with Uptravi in the trial include headache, diarrhea, jaw pain, nausea, muscle pain (myalgia), vomiting, pain in an extremity, and flushing.

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