Baxter Submits Application for U.S. FDA Approval of BAX111, Investigational Recombinant Treatment for Von Willebrand Disease

From Baxter:

Baxter Submits Application for U.S. FDA Approval of BAX111, Investigational Recombinant Treatment for Von Willebrand Disease  

DEERFIELD, Ill., DECEMBER 22, 2014 - Baxter International Inc. (NYSE:BAX) today announced that the company has submitted a biologics license application (BLA) to the United States (U.S.) Food and Drug Administration (FDA) for the approval of BAX111, the first highly-purified recombinant von Willebrand Factor (rVWF) in clinical development as a treatment for patients with von Willebrand disease, the most common type of inherited bleeding disorder.¹

"If approved, BAX111 will be the first recombinant replacement treatment for von Willebrand disease, offering an important new option that may provide greater flexibility in treating patients with this challenging disease," said John Orloff, vice president and global head of research and development at Baxter BioScience. "Filing for U.S. approval for this treatment helps us further advance our pursuit of new treatment options and improved quality of care for people with a range of bleeding disorders around the world."

The filing was based on the completion of a Phase III, multi-center, open-label clinical trial assessing the safety, efficacy and pharmacokinetics of BAX111. The study met its primary efficacy endpoint defined by the number of patients who achieved treatment success for control of bleeding episodes. All patients treated in the full analysis set (n=22) experienced a 100% treatment success rating based on a 4-point efficacy rating scale, comparing prospectively estimated number of infusions needed to treat the bleeding episodes to the actual number of infusions administered. The median number of infusions required to treat bleeding events in the trial was 1.0 and the majority of events (81.1%) were resolved with a single infusion.

A total of 125 adverse events (AE's) following 318 infusions occurred in 25/37 subjects. Eight AEs were considered causally related to BAX111: six non-serious related AEs (tachycardia, infusion site paresthesia, electrocardiography (ECG) T-wave inversion, dysgeusia, generalized pruritis and hot flush) occurred in four patients, and two related SAEs (chest discomfort and increased heart rate) occurred in one patient.

Baxter expects to publish additional data from the trial in the coming months. Both the European Commission and the U.S. Food and Drug Administration have granted orphan-drug designation for BAX111.

About von Willebrand Disease ^2,3
Von Willebrand Disease (VWD) is an autosomal genetic disorder related to quantitative deficits and/or qualitative defects of VWF, the result of which is impaired hemostasis. It is the most common hereditary coagulation disorder, occurring in approximately one to two percent of the general population. Many people who have VWD may experience mild symptoms, but some patients can experience severe bleeding events similar to bleeding experienced by patients with hemophilia.

About Baxter BioScienceBaxter BioScience is a leading provider of therapeutic treatments that save, sustain and improve the lives of people with rare conditions, chronic diseases or limited treatment options. Supported by advanced technical and manufacturing expertise, Baxter BioScience has a broad pipeline built on a legacy of innovation in bleeding disorders and immunology and is expanding to address emerging opportunities in niche areas of oncology as well as technology platforms such as biosimilars.

About Baxter International Inc.Baxter International Inc., through its subsidiaries, develops, manufactures and markets products that save and sustain the lives of people with hemophilia, immune disorders, cancer, infectious diseases, kidney disease, trauma and other chronic and acute medical conditions. As a global, diversified healthcare company, Baxter applies a unique combination of expertise in medical devices, pharmaceuticals and biotechnology to create products that advance patient care worldwide.

This release includes forward-looking statements concerning BAX111, including expectations with regard to the potential impact of BAX111 to patients. The statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; additional clinical results; changes in laws and regulations; product quality, manufacturing or supply, or patient safety issues; and other risks identified in Baxter's most recent filing on Form 10-K and other SEC filings, all of which are available on Baxter's website. Baxter does not undertake to update its forward-looking statements.

¹Laffan MA, Lester W, O'Donnell JS, et al. The diagnosis and management of von Willebrand disease: a United Kingdom Haemophilia Centre Doctors Organization guideline approved by the British Committee for Standards in Haematology. Br J Haematol. 2014; 167: 453-465.
²Nichols WL, Hultin MB, James AH, et al. Guidelines. von Willebrand disease (VWD): evidence-based diagnosis and management guidelines, the National Heart, Lung, and Blood Institute (NHLBI) Expert Panel report (USA). Haemophilia. 2008; 14: 171-232.
³Mannucci PM. Treatment of von Willebrand's disease. N Eng J Med. 2004; 351: 683-694.