Actelion Pharmaceuticals announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) seeking to expand the indication of OPSUMIT (macitentan) to include the treatment of adults with inoperable chronic thromboembolic pulmonary hypertension (CTEPH, WHO Group 4) to improve exercise capacity and pulmonary vascular resistance (PVR).
The filing is based on data from the MERIT-1 trial, which showed significant improvements in the primary and secondary endpoint of PVR and 6MWD, respectively, compared with background therapy. OPSUMIT was well tolerated in this patient population and safety was generally consistent with the known safety profile for OPSUMIT from previous clinical studies. The study results were presented at the American Thoracic Society (ATS) 2017 and European Respiratory Society (ERS) 2017 and published in The Lancet Respiratory Medicine.
A substantial proportion (almost 40%) of CTEPH patients are deemed inoperable and current pharmacological treatment options for these patients are limited to one therapy only. MERIT-1 provides the first randomized controlled trial data involving patients on combination therapy, addressing a key unmet need in CTEPH.
"Inoperable chronic thromboembolic pulmonary hypertension is associated with a poor prognosis if left untreated. At Actelion, we are committed to advancing therapies for pulmonary hypertension patients around the world and we believe that this application may provide a new therapeutic option for those living with inoperable CTEPH in the United States. With this latest milestone, Actelion is continuing to build on its 20-year legacy of ground-breaking innovation in this life-limiting disease," Martin Fitchet, M.D., Global Head, Research & Development at Actelion Pharmaceuticals said.
OPSUMIT is an orally active endothelin receptor antagonist (ERA) that is currently approved in the U.S. for the treatment of PAH (WHO Group I) to delay disease progression and hospitalization.