United Therapeutics Corporation and its wholly-owned subsidiary, United Therapeutics Europe, Ltd., announced that the French regulatory agency Agence Francaise de Securite Sanitaire des Produits de Sante (AFSSAPS) has approved intravenous use of Remodulin (treprostinil) for the treatment of pulmonary arterial hypertension (PAH).
Remodulin is already approved in most of Europe for the continuous subcutaneous infusion treatment of idiopathic or heritable PAH to improve exercise tolerance and symptoms of the disease in patients classified as New York Heart Association (NYHA) functional class III. The AFSSAPS' approval follows a review period during which 22 European member nations, each of which had previously approved subcutaneous Remodulin through the mutual recognition process, reviewed and endorsed the final variation assessment report (FVAR) issued by AFSSAPS, which will allow the use of the intravenous route of delivery to the Remodulin in the labelling in those nations.
“It is with great satisfaction that intravenous use of Remodulin will now be available to PAH patients in Europe,” said Roger A Jeffs, PhD, president and chief operating officer. “This approval significantly expands the treatment options for PAH patients using parenteral therapy in Europe.”
In Europe, risk management plans (RMPs) are routinely required as part of the regulatory approval process for new medicines and also for significant variations involving a change to the route of administration, formulation or indication. For intravenous Remodulin, United Therapeutics will implement an RMP focused on minimizing the known risks of central venous catheter-related blood stream infections associated with intravenous administration.
United Therapeutics also informed that it has submitted to the US Food and Drug Administration a New Drug Application (NDA) for treprostinil diethanolamine sustained release tablets (oral treprostinil) for the treatment of PAH on December 27, 2011. The submission starts a 60-day period during which the FDA will examine the application for completeness. If the FDA accepts the NDA for review, then it is expected to be subject to the standard review period of 10 months from the submission date, before an action letter is issued.
Remodulin is a prostacyclin vasodilator indicated in the United States for the treatment of pulmonary arterial hypertension (PAH) (WHO Group 1) to diminish symptoms associated with exercise. Studies establishing effectiveness included patients with NYHA Functional Class II-IV symptoms and etiologies of idiopathic or heritable PAH (58%), PAH associated with congenital systemic-to-pulmonary shunts (23%), or PAH associated with connective tissue diseases (19%). Remodulin may be administered as a continuous subcutaneous infusion or continuous intravenous infusion; however, because of the risks associated with chronic indwelling central venous catheters, including serious blood stream infections, continuous intravenous infusion should be reserved for patients who are intolerant of the subcutaneous route, or in whom these risks are considered warranted.
In patients with PAH requiring transition from Flolan (epoprostenol sodium), Remodulin is indicated to diminish the rate of clinical deterioration. The risks and benefits of each drug should be carefully considered prior to transition.
Chronic intravenous infusions of Remodulin are delivered using an indwelling central venous catheter. This route is associated with the risk of blood stream infections, or BSI, and sepsis, which may be fatal. Therefore, continuous subcutaneous infusion is the preferred mode of administration.
United Therapeutics Corporation is a biotechnology company focused on the development and commercialization of unique products to address the unmet medical needs of patients with chronic and life-threatening cardiovascular and infectious diseases and cancer.