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FDA Approves Baxter's OBIZUR [Antihemophilic Factor (Recombinant), Porcine Sequence], for Acquired Hemophilia A

Approval Based on Data Showing 100% of 28 Patients Responded to Treatment within 24 Hours

DEERFIELD, Ill., October 24, 2014 - Baxter International Inc. (NYSE:BAX) today announced that the United States Food and Drug Administration (FDA) has approved OBIZUR [Antihemophilic Factor (Recombinant), Porcine Sequence] for the treatment of bleeding episodes in adults with acquired hemophilia A (AHA), a very rare and potentially life-threatening acute bleeding disorder. OBIZUR was granted orphan-drug status by the FDA and its review was prioritized based on AHA's classification as a rare disease and the potential for the treatment to address an important unmet medical need.

OBIZUR is the first recombinant porcine FVIII treatment approved for AHA that allows physicians to manage the treatment's efficacy and safety by measuring factor VIII activity levels in addition to clinical assessments. OBIZUR replaces the inhibited human factor VIII with a recombinant porcine sequence factor VIII based on the rationale that it is less susceptible to inactivation by circulating human factor VIII antibodies.

"The approval of OBIZUR is welcome news for the hemophilia community based on the data from the first clinical trial designed specifically for acquired hemophilia A, which found that all patients responded to treatment within 24 hours," said Dr. Rebecca Kruse-Jarres, Director of the Hemophilia Care Program at Puget Sound Blood Center in Seattle and the clinical trial's principal investigator. "Importantly, this new option to treat bleeding episodes will enable us to measure factor VIII levels, thus giving us an objective marker of hemostasis that can guide dosing and prevent overdosing."

The approval is based on a global, prospective, controlled, multi-center Phase 2/3 open-label clinical trial that examined the efficacy of OBIZUR in the treatment of serious bleeding episodes in adults with AHA (29 patients evaluated for safety, 28 evaluated for efficacy). All patients treated with OBIZUR (28/28) showed a positive response, meaning an effective or partially effective response with bleeding stopped or reduced and clinical improvement, at
24 hours after the initial infusion. A total of 86 percent (24/28) had successful treatment of the initial bleeding episode. The overall treatment success was determined by the investigator based on the ability to discontinue or reduce the dose and/or dosing frequency of OBIZUR. Common adverse reactions observed in greater than five percent of 29 patients in the clinical trial were development of inhibitors to porcine FVIII.

"As a new treatment option with the ability to measure FVIII activity in the body, OBIZUR will address important unmet needs for patients with acquired hemophilia A, a potentially life-threatening condition," said Brian Goff, head of Baxter's hemophilia franchise. "This approval reflects Baxter's long-standing commitment to discovering new options for hemophilia patients and adds to our portfolio of treatments that reduce the burden of these diseases."

OBIZUR will be commercially available in the United States in the coming months and is currently under regulatory review in Europe and Canada.

About OBIZUR
OBIZUR [Antihemophilic Factor (Recombinant), Porcine Sequence] is indicated for the treatment of bleeding episodes in adults with acquired hemophilia A.

Limitations of Use:

  • Safety and Efficacy of OBIZUR has not been established in patients with baseline anti-porcine factor VIII inhibitor titer greater than 20 BU.
  • OBIZUR is not indicated for the treatment of congenital hemophilia A or von Willebrand disease.


Detailed Important Risk Information for OBIZUR

CONTRAINDICATIONS
OBIZUR is contraindicated in patients who have had life-threatening hypersensitivity reactions to OBIZUR or its components (including traces of hamster proteins).

WARNINGS & PRECAUTIONS

Hypersensitivity Reactions
Hypersensitivity reactions can occur with OBIZUR. OBIZUR contains trace amounts of hamster proteins. Early signs of allergic reactions, which can progress to anaphylaxis, include angioedema, chest-tightness, dyspnea, hypotension, wheezing, urticaria, and pruritus. Immediately discontinue administration and initiate appropriate treatment if allergic or anaphylactic-type reactions occur.

Inhibitory Antibodies
Inhibitory antibodies to OBIZUR have occurred. Monitor patients for the development of antibodies to OBIZUR by appropriate assays. If the plasma factor VIII level fails to increase as expected, or if bleeding is not controlled after OBIZUR administration, suspect the presence of an anti-porcine factor VIII antibody. If such inhibitory antibodies to anti-porcine factor VIII are suspected and there is a lack of clinical response, consider other therapeutic options.

Monitoring Laboratory Tests

  • Perform one-stage clotting assay to confirm that adequate factor VIII levels have been achieved and maintained
    • Monitor factor VIII activity 30 minutes and 3 hours after initial dose
    • Monitor factor VIII activity 30 minutes after subsequent doses
  • Monitor the development of inhibitory antibodies to OBIZUR. Perform a Nijmegen Bethesda inhibitor assay if expected plasma factor VIII activity levels are not attained or if bleeding is not controlled with the expected dose of OBIZUR. Use Bethesda Units (BU) to report inhibitor levels.


ADVERSE REACTIONS
Common adverse reactions observed in greater than 5% of subjects in the clinical trial were development of inhibitors to porcine factor VIII.

Please see full prescribing information for OBIZUR at: www.baxter.com/downloads/healthcare_professionals/products/OBIZUR_PI.pdf.

About Acquired Hemophilia A
Acquired hemophilia A is a rare, potentially life-threatening bleeding disorder, which, unlike congenital hemophilia, typically affects older adults and occurs in both males and females. In acquired hemophilia A, individuals typically experience subcutaneous, soft tissue, and post-surgical bleeding. The comorbidities in this typically elderly population also pose a particular challenge to treat serious bleeding episodes.

About Baxter in Hemophilia
Baxter has more than 60 years experience in hemophilia and has introduced a number of therapeutic firsts for hemophilia patients. Baxter has the broadest portfolio of hemophilia treatments in the industry and is able to meet individual therapy choices, providing a range of options at each treatment stage. The company's work is focused on optimizing hemophilia care and improving the lives of people living with hemophilia A and B, as well as acquired hemophilia A, worldwide.

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 OBIZUR, including expectations with regard to its potential impact on patients, commercial launch plans in the US and planned regulatory filings outside the US. 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; product quality or supply or patient safety issues; changes in law and regulations; 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.