Opsumit wins US approval
pharmafile | October 22, 2013 | News story | Sales and Marketing | actelion fda, opsumit
The FDA has approved Actelion’s oral endothelin receptor antagonist Opsumit as a treatment for pulmonary arterial hypertension (PAH), a potentially fatal condition.
Opsumit (macitentan) – which is also currently being investigated in patients with the most aggressive form of brain cancer, glioblastoma (GBM) – is the only oral PAH therapy which has been proven to delay disease progression.
The condition is characterised by abnormally high blood pressure in the arteries between the heart and lungs – and in the Phase III SERAPHIN study, Opsumit 10mg met its primary endpoint, reducing the risk of the first occurrence of a morbidity or mortality event by 45% compared to placebo.
The results, published in the New England Journal of Medicine in August, showed that this effect held whether patients had received other PAH therapies or not.
Opsumit also demonstrated a risk reduction in PAH-related hospitalisation and death of 50% compared to placebo.
SERAPHIN is the largest PAH outcome study to date, with Opsumit given on average for two years as monotherapy, or in combination with phosphodiesterase-5 inhibitors or inhaled prostanoids.
Patients taking part had idiopathic and heritable PAH (57%), PAH caused by connective tissue disorders (31%) and PAH caused by congenital heart disease with repaired shunts (8 per cent).
“Over the past 20 years, great strides have been made in treating PAH patients,” said Dr Vallerie McLaughlin, director of the Pulmonary Hypertension Programme in the Division of Cardiovascular Medicine at the University of Michigan.
“However, there has been a medical need for innovative treatments that improve long-term outcomes. Opsumit is the first clinically proven and only oral treatment option indicated to delay disease progression and reduce the need for PAH hospitalisation,” McLaughlin said.
PAH has any number of symptoms, ranging from slight breathlessness to the appearance of heart failure – and the condition can severely reduce life expectancy.
For the purposes of Opsumit’s indication, disease progression includes the need to start using prostanoids, clinical worsening of PAH – shown by decreased ability to walk or the need for additional PAH treatment – and, of course, death.
Actelion chief executive Jean-Paul Clozel said: “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.”
Adam Hill
