Opsumit is indicated for the treatment of pulmonary arterial hypertension (PAH, WHO Group 1) to delay disease progression. Disease progression included: death, initiation of intravenous (IV) or subcutaneous prostanoids or clinical worsening of PAH (decreased 6-minute walk distance, worsened PAH symptoms and need for additional PAH treatment). Opsumit also reduced hospitalization for PAH.  

 

Effectiveness was established in a long-term study in PAH patients with predominantly WHO Functional Class 2-3 symptoms treated for an average of two years. Patients were treated with Opsumit monotherapy or in combination with phosphodiesterase-5 inhibitors or inhaled prostanoids. Patients had idiopathic and heritable PAH (57%), PAH caused by connective tissue disorders (31%) and PAH caused by congenital heart disease with repaired shunts (8%).

 

Dr. Vallerie McLaughlin, director of the Pulmonary Hypertension Program in the Division of Cardiovascular Medicine at the University of Michigan, commented: “Over the past twenty years, great strides have been made in treating PAH patients. 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 hospitalization.”

 

McLaughlin concluded: “These effects were demonstrated in SERAPHIN, the first and largest PAH outcome study to date, where Opsumit was given on average for two years, as a monotherapy or in combination with phosphodiesterase-5 inhibitors or inhaled prostanoids. I am very pleased that PAH patients will have this new treatment option.”

 

Jean-Paul Clozel, MD and CEO of Actelion commented: “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. Over the last 14 years, Actelion has worked tirelessly to first discover and then develop Opsumit in the largest, longest and first-ever outcome study in PAH. 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. We will now leverage our existing PAH expertise and infrastructure to bring Opsumit to patients within the coming weeks.”    

 

The U.S. label for Opsumit carries a boxed warning alerting patients and health care professionals that the drug should not be used in pregnant women because it can harm the developing fetus. Female patients can receive the drug only through the Opsumit REMS Program. All female patients must be enrolled in the program, comply with pregnancy testing requirements and be counselled regarding the need for contraception.

 

The most common adverse reactions (more frequent than placebo by 3% or more) observed in patients treated with Opsumit were anemia, nasopharyngitis/pharyngitis, bronchitis, headache, influenza, and urinary tract infection.

 

In the United States, Actelion expects Opsumit to become available to patients in November. Outside of the United States, Actelion continues to work with health authorities to obtain regulatory approval for Opsumit .

 

Date: October 21, 2013

Source: Actelion Ltd.