Actelion Pharmaceuticals: FDA Approves Orphan Drug In Time For Awareness Month
Just in time for November’s Pulmonary Hypertension Awareness Month, the FDA on October 18, approves Actelion Pharmaceuticals’ orphan drug Opsumit (Macitentan) for the rare disease Pulmonary Arterial Hypertension (PAH). Opsumit is the 11th PAH treatment approved by the FDA. The approval of Opsumit comes just 10 days after the FDA October 8 approval of Bayer Healthcare’s orphan drug Adempas (Riociguat) for 2 forms of pulmonary hypertension :
Both drugs are the first in 5 years to be approved by the FDA for PAH.
A patient with PAH has high blood pressure in arteries that supply the lungs; the muscles in the walls of the arteries thicken and the arteries in the lungs get narrower. This results in difficulty of the blood flowing to the lungs. PAH is debilitating and life-threatening, shortening the life of a patient due to breathing problems and possible heart failure. There is currently no cure for PAH.
Actelion Pharmaceuticals’ Opsumit is an oral, once daily, endothelin receptor antagonist approved for PAH, to delay disease progression. Opsumit :
• Receives FDA Orphan Drug Designation (ODD) for PAH in September 2009
• Receives FDA ODD for Idiopathic Pulmonary Fibrosis in April 2009
• Receives European orphan designation in September 2011 for PAH
• Receives European orphan designation in January 2010 for Idiopathic Pulmonary Fibrosis.
The FDA approval is based in part on data from a Phase III SERAPHIN study that is published in the August 2013 journal, New England Journal of Medicine :
Pulido T, Adzerikho I, Channick RN, Delcroix M, Galiè N, Ghofrani HA, Jansa P, Jing ZC, Le Brun FO, Mehta S, Mittelholzer CM, Perchenet L, Sastry BK, Sitbon O, Souza R, Torbicki A, Zeng X, Rubin LJ, Simonneau G; SERAPHIN Investigators. Macitentan and morbidity and mortality in pulmonary arterial hypertension. N Engl J Med. 2013 Aug 29;369(9):809-18. doi: 10.1056/NEJMoa1213917.
The SERAPHIN Phase III study shows that the primary endpoint, risk of the first occurrence of a morbidity or mortality event, is reduced by 45 %.
Per Jean-Paul Clozel, M.D., CEO of Actelion Pharmaceuticals:
“Today’s approval of Opsumit by the FDA is providing the PAH community with a unique treatment option, the only oral PAH medicine that has proven to delay disease progression … I would like to express my gratitude to all the members of the PAH community. Without their contribution, Opsumit would not have become a reality …”.
In the United States, Actelion Pharmaceuticals expects Opsummit to become available in November. Outside of the United States, Actelion Pharmaceuticals is working with health authorities to obtain regulatory approval for Opsumit.