Tracleer and PPH News FDA Approvals: Ventavis, Kepivance, Captique Yael Waknine Jan. 5, 2005 — The U.S. Food and Drug Administration (FDA) has approved iloprost inhalation solution for the treatment of severe pulmonary arterial hypertension, palifermin injection to reduce the incidence and duration of severe oral mucositis secondary to chemotherapy and radiation therapy in patients with hematologic malignancies, and a hyaluronic acid-based dermal filler for the correction of moderate to severe facial wrinkles. Iloprost Inhalation Solution (Ventavis) for Severe Pulmonary Arterial Hypertension On Dec. 29, the FDA approved iloprost inhalation solution (Ventavis, made by CoTherix, Inc.), for the treatment of pulmonary arterial hypertension (PAH) in patients with New York Heart Association (NYHA) Class III or IV symptoms. The synthetic prostacyclin analog is intended for administration with Prodose Adaptive Aerosol Delivery (AAD) system. According to a company news release, the non-invasive nature of the system is expected to increase patient compliance and avoid complications associated with intravenous or subcutaneous prostacyclin delivery. The approval was based on the results of a controlled trial comparing the effects of repeated daily inhalations of iloprost (2.5- or 5.0-micrograms 6 to 9 times daily) with placebo in 203 adult patients with selected forms of severe PAH and chronic thromboembolic pulmonary hypertension, NYHA functional class III or IV. The median inhaled dose of iloprost was 30 micrograms/day. At three months, 16.8% of patients receiving iloprost showed significant improvement in symptoms (at least one NYHA class), and exercise tolerance (minimum 10% increase in six-minute walking distance), compared with 4.9% of patients receiving placebo (P = .0033). Iloprost was generally well tolerated. The most commonly reported adverse events included flushing, increased cough, hypotension, headaches, nausea, trismus, and syncope. Iloprost inhalation solution was previously approved for this indication in several European countries and Australia, where it is marketed by Schering AG. Palifermin (Kepivance) Reduces Incidence and Duration of Severe Mucositis On Dec. 15, the FDA approved palifermin (Kepivance, made by Amgen Inc.), to decrease the incidence and duration of severe oral mucositis in patients with hematologic malignancies undergoing chemotherapy with or without radiation in preparation for bone marrow transplants. It is the first drug to be approved for this indication by the FDA. Palifermin is a recombinant human keratinocyte growth factor (KGF) that stimulates epithelial cell growth in areas damaged by chemotherapy and/or radiation, such the lining of the mouth and digestive tract. The approval was based on the results of a phase 3 double-blind trial comparing use of palifermin 60 mcg/kg/day administered intravenously for three days prior to conditioning therapy (fractionated total-body irradiation plus high-dose chemotherapy) and repeated after bone marrow transplantation, with placebo. Palifermin therapy was found to significantly reduce the incidence of World Health Organization (WHO) grade 4 and grade 3-4 mucositis, relative to placebo (20% vs. 62% and 63% vs. 98%, respectively; P < .001 for both). Duration of WHO grade 2-4 painful oral mucositis was likewise significantly reduced in patients receiving palifermin (8 days vs. 14 days with placebo). Palifermin was found to significantly decrease mouth and throat soreness (P < .001) and improve patients' ability to eat, drink, swallow, and talk. Fewer days of morphine were required for pain relief in patients administered palifermin, relative to placebo (7 days vs. 11 days). Adverse events were transient and mild to moderate in severity, and included rash, pruritis, erythema, paresthesias, mouth/tongue disorders, and taste alteration. The FDA notes that the safety and efficacy of palifermin has not been established in patients with non-hematologic malignancies. Dermal Filler (Captique) for Moderate to Severe Facial Wrinkles On Dec. 2, the FDA approved a dermal filler (Captique Injectable gel, made by Genzyme and marketed by Inamed Corporation), for the correction of moderate to severe facial wrinkles. According to a company news release, the product is the first non-animal stabilized hyaluronic acid-based dermal filler to be approved in the United States. The clear, colorless gel adds volume to correct moderate to severe facial wrinkles and folds around the nose and mouth with immediate results that last up to one year. According to the manufacturer, the results, characteristics, and safety profile of the product are similar to that of Restylane (made by Q-Med AB), that was approved by the FDA in 2003.
Reviewed by Gary D. Vogin, MD
United Therapeutics subsidiary finishes enrollment for cancer drug trials
Washington Business Journal
by Neil AdlerStaff Reporter
June 21, 2006 - A subsidiary of United Therapeutics has completed enrollment in two pivotal trials for a drug to treat ovarian cancer.
Enrollment in the first trial for the drug, called OvaRex, was completed in December 2005 and this week the second trial reached its enrollment goal of 177 patients.
Unither Pharmaceuticals, a wholly owned subsidiary of Silver Spring-based United Therapeutics (NASDAQ: UTHR), is coordinating the trials, which are being conducted at more than 60 centers throughout the United States.
OvaRex is a monoclonal antibody, a type of biotech drug, in development for the treatment of stage III/IV advanced ovarian cancer. The drug targets a protein expressed in most ovarian cancers, and is designed to help patients' immune systems recognize and more effectively fight the cancer.
Ovarian cancer is the fifth leading cause of cancer deaths among women in the United States.
The American Cancer Society estimates that this year alone more than 20,000 new cases of ovarian cancer will be diagnosed and more than 15,000 women will die from this type of cancer.
United Therapeutics has built a roughly 50,000-square-foot lab near its headquarters that will produce materials used in both Remodulin, the company's lead drug, and OvaRex.
United Therapeutics, one of several public Washington-area biotech firms to have a product on the market and be in the black, develops drugs for cancer, cardiovascular disorders and infectious diseases.
Study bodes well for Tracleer use in IPF - Actelion
ZURICH, May 24 (Reuters) - Actelion (ATLN.S: Quote, Profile, Research) said new data give strong reason to further evaluate its flagship drug Tracleer in a phase III study for use in patients with idiopathic pulmonary fibrosis (IPF).
The Swiss biotech firm said late on Tuesday a trial showed 22.5 percent of patients on Tracleer either showed disease progression or died within 12 months.
That represented a relative risk reduction of 38 percent compared compared with a placebo group, where the rate was 36.1 percent.
There are currently no approved therapies for IPF, a disease with median survival of patients of less than three years.
Actelion's Tracleer, used to treat pulmonary arterial hypertension, had sales of 201.4 million Swiss francs ($166.7 million) in the first quarter of the year.
Analysts say that with competition for Tracleer increasing, the company must seek other uses for the drug beyond the rare disease and the firm is therefore spending heavily on research and development on a pipeline of new compounds.
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