Actelion Ltd, a biopharmaceutical company, announced that it has submitted a new drug application (NDA) for Tracleer for the treatment of pulmonary arterial hypertension (PAH) with the Japanese health authorities.
Tracleer, an orally available dual endothelin receptor antagonist, is already available in key markets worldwide, including the United States, the European Union, Canada and Switzerland. In Australia, the drug has been approved and the reimbursement review is ongoing.
The NDA in Japan also reports - in addition to the two registration studies already published - three successfully concluded bridging studies, indicating that the drug shows comparable safety and efficacy in Japanese patients.
Dr. Satoshi Tanaka, president Actelion Pharmaceuticals Japan Ltd, commented: "I am very pleased that our investment in a fully-fledged Actelion organization in Japan has allowed us to rapidly translate and adapt the original NDA. We are fully prepared to answer any questions the Japanese health authorities might have in the upcoming review process."
In Japan, Tracleer has been granted Orphan Drug status earlier in the year. This allows the Japanese health authorities to review the NDA applying a fast track procedure. Accordingly, Actelion Japan already employs 25 Japanese professionals in the areas of Drug Development, Drug Safety, Drug Regulatory Affairs and Quality Assurance. The company has also started to hire additional personnel in Marketing.
In line with the progress of the NDA review, Actelion Japan will complete its sales force organization to fully serve the Japanese PAH market.
Approximately 100,000 people in Europe and the United States are afflicted with either primary pulmonary arterial hypertension or secondary forms of the disease related to conditions or tissue disorders that affect the lungs, such as scleroderma, lupus, HIV/AIDS, congenital heart disease or the use of certain appetite suppressants.
The first signs of the disease, such as mild shortness of breath, fatigue and difficulty exercising, are so subtle that the disease is often either misdiagnosed or not diagnosed until the patient's condition is far advanced. The survival rate for PAH in untreated patients is only 40 to 55 per cent at two years from the onset of symptoms. Once patients reach more advanced stages of PAH (WHO Class III and IV), they often have no choice but to go on epoprostenol sodium therapy, which requires a 24-hour infusion pump and an intravenous line implanted through the chest directly into the patient's heart. Ultimately, many patients require lung transplantation.
In the two pivotal clinical studies, approximately 11 per cent of PAH patients receiving Tracleer experienced abnormal but reversible liver enzyme elevations. It is therefore important that patients undergo monthly liver monitoring. Due to the risk of birth defects, women who are pregnant, or woman of childbearing age who do not use a reliable method of contraception, must not take Tracleer.