Actelion Pharmaceuticals US, Inc., announced that the U.S. Food and Drug Administration (FDA) has approved the brand name VELETRI® for the company's epoprostenol for injection therapy. VELETRI has been commercially available since April 2010 as Epoprostenol for Injection for the treatment of moderate to severe pulmonary arterial hypertension (PAH) and PAH associated with the scleroderma spectrum of disease. Actelion plans to release VELETRI-labeled product by early fourth quarter of 2010.

VELETRI is an improved formulation of epoprostenol that offers greater convenience to patients than other epoprostenol formulations. VELETRI is stable at room temperature for up to 24 hours when diluted as directed and put into the pump for administration, eliminating the need for ice packs.

"VELETRI is a proven therapy for the treatment of PAH patients who don't respond adequately to conventional treatment, and rounds out a portfolio of therapies designed to address the diverse needs of patients with this chronic and life-threatening disease," said Shal Jacobovitz, president of Actelion Pharmaceuticals US, Inc. "As the pioneer in PAH, we are committed to transforming the lives of patients by developing efficacious therapies that also offer flexibility and convenience in treatment."

In conjunction with the launch of VELETRI, Actelion opened its fourth PAH patient registry in the United States. PROSPECT, the registry to PROSPECTively evaluate use of VELETRI in patients with PAH, is a multicenter, observational, U.S.-based registry, which is currently ongoing.

Actelion will also provide further information on VELETRI at the American Heart Association Scientific Sessions 2010 in Chicago with a poster entitled "Biocomparability of Two Formulations of Epoprostenol, Epoprostenol for Injection (ACT-385781A) And Flolan®, Via Pharmacokinetic Assessment of Two Primary Metabolites."

The registration process for Epoprostenol for Injection is ongoing outside the US, initially in France, also with VELETRI as proposed brand name.

About VELETRI®

VELETRI is indicated for the long-term intravenous treatment of primary pulmonary hypertension and pulmonary hypertension associated with the scleroderma spectrum of disease in NYHA Class III and Class IV patients who do not respond adequately to conventional therapy. Unlike other epoprostenol formulations approved for PAH, VELETRI is stable at room temperature (77 F, 25 C), for up to 24 hours after it has been diluted, making the use of frozen gel packs unnecessary. VELETRI can be reconstituted with either Sterile Water for Injection, USP, or Sodium Chloride 0.9% Injection, USP, eliminating the need for drug-specific diluents.

Accredo Health Group, Inc. is the sole specialty pharmacy provider of VELETRI. Accredo offers an enhanced level of personalized service to patients with chronic and complex disease, and will provide call center, nursing and reimbursement support services for patients using VELETRI and their healthcare providers.

About Pulmonary Arterial Hypertension (PAH)

Pulmonary arterial hypertension (PAH) is a chronic, life-threatening disorder characterized by abnormally high blood pressure in the arteries between the heart and lungs of an affected individual. The function of the heart and lungs is severely compromised, manifested by a limited exercise capacity, and, ultimately, a reduced life expectancy. Approximately 100,000 people in Europe and the United States are afflicted with either primary or secondary forms of the disease related to conditions or tissue disorders that affect the lungs, such as scleroderma, lupus, HIV/AIDS or congenital heart disease.

PAH is associated with structural changes in both the pulmonary vasculature and the right ventricle. Recent advances [1] in the understanding of the pathogenic factors leading to the pulmonary vascular disease have led to the development of new therapies targeting specific pathways (the prostacyclin pathway; the endothelin pathway; and the nitric oxide pathway) (2). The available therapies have positive effects in PAH, but they do not provide a cure, and in many patients the disease will progress. PAH remains a serious life-threatening condition (2,3). Early recognition and an understanding of the selection and timing of therapeutic options remain critical elements in the optimal management of patients with this disorder.

References

(1) Farber HW; Loscalzo J. Mechanisms of disease: pulmonary arterial hypertension. N. Eng. J. Med. 2004; 351:1655-65.

(2) Humbert M; Sitbon O; Simonneau G. Treatment of pulmonary arterial hypertension. N. Eng. J. Med. 2004; 351:1425-36.

(3) Humbert M; Morrell NW; Archer SL; et al. Cellular and molecular pathobiology of pulmonary arterial hypertension. J. Am. Coll. Cardiol. 2004; 43: Suppl. 12: 13S-24S.

Source: Actelion Pharmaceuticals US, Inc