BANNOCKBURN, Ill.--(BUSINESS WIRE)--Baxalta Incorporated (NYSE: BXLT), a global biopharmaceutical leader dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, today announced that Health Canada has approved OBIZUR [Antihemophilic Factor (Recombinant), Porcine Sequence] for the treatment of bleeding episodes in patients with acquired hemophilia A caused by autoantibodies to coagulation Factor VIII (FVIII), a very rare and life-threatening acute bleeding disorder. OBIZUR is the first recombinant porcine sequence FVIII treatment available for patients with acquired hemophilia A, specifically designed to enable physicians to monitor treatment response by measuring FVIII activity levels in addition to clinical assessments.
“The approval of OBIZUR in Canada provides physicians with an important new and effective treatment for acquired hemophilia A," said Dr. Jean St-Louis, hematologist at Hôpital Maisonneuve-Rosemont and associate professor at Université de Montréal. "This is a very challenging condition to treat and as an investigator in the clinical study, I had the opportunity to see the positive impact that OBIZUR can have for patients and their treating team.”
The approval is supported by the positive results of a global, prospective, controlled, multi-center Phase II/III open-label clinical trial that examined the efficacy of OBIZUR in the treatment of serious bleeding episodes in patients with acquired hemophilia A. All patients treated with OBIZUR, 100 percent (28/28), showed a positive response and clinical improvement at 24 hours after the initial infusion. Successful control (resolution) of the initial bleeding episode was observed in 86 percent (24/28) of all patients and in 94 percent (16/17) of those treated with OBIZUR as the first-line treatment. The development of antibodies to porcine FVIII was the only adverse reaction reported in more than five percent of patients.1
“As the first recombinant porcine option for acquired hemophilia in Canada, OBIZUR offers a significant advancement for physicians treating this challenging disease by allowing them to monitor levels of FVIII activity and adjust based on their patient’s response to the treatment,” said Brian Goff, executive vice president and president, Hematology, Baxalta. “We look forward to introducing this treatment for the hemophilia community in Canada and continuing our efforts to reduce the burden of bleeding disorders as we pursue a world without bleeds.”
OBIZUR is now approved in the United States and Canada, and is under regulatory review in the European Union, Switzerland, Australia and Colombia.
About OBIZUR in Canada
OBIZUR [Antihemophilic Factor (Recombinant), Porcine Sequence] is indicated for the treatment of bleeding episodes in patients with acquired hemophilia A.
CONTRAINDICATIONS
OBIZUR is contraindicated in patients with known anaphylactic reactions to the following: the active substance, any ingredient in the formulation, hamster protein or any component of the container.
WARNINGS & PRECAUTIONS
General
Allergic type hypersensitivity reactions (including anaphylaxis) may occur. The product contains trace amounts of hamster proteins. Early signs of allergic reactions, which can progress to anaphylaxis, include angioedema, chest-tightness, hypotension, lethargy, nausea, vomiting, paresthesia, restlessness, wheezing, and dyspnea. Immediately discontinue administration and initiate appropriate treatment if allergic or anaphylactic-type reactions occur.
Inhibitors
Inhibitory antibodies to OBIZUR have occurred in patients treated with OBIZUR (see ADVERSE REACTIONS). 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 an anti-porcine factor VIII antibody can be suspected. An adjustment of the dose or other therapeutic options should be considered.
ADVERSE REACTIONS
The most frequently reported Adverse Drug Reactions (ADRs) included constipation, diarrhea, hypokalemia, anemia, oedema peripheral and a positive anti-porcine inhibitor test result.
Non-serious Adverse Reactions occurred in 6.9% of subjects, these 2 subjects developed anti-porcine FVIII inhibitors.
About Acquired Hemophilia A
Acquired hemophilia A is a rare, life-threatening bleeding disorder, which, unlike congenital hemophilia, typically affects older adults and occurs in both males and females.2,3 In acquired hemophilia A, individuals typically experience subcutaneous, soft tissue, and post-surgical bleeding.3,4,5 The comorbidities in this typically elderly population also pose a particular challenge to treat serious bleeding episodes.2
About Baxalta
Baxalta Incorporated (NYSE: BXLT) is a $6 billion global biopharmaceutical leader developing, manufacturing and commercializing therapies for orphan diseases and underserved conditions in hematology, oncology and immunology. Driven by passion to make a meaningful impact on patients’ lives, Baxalta’s broad and diverse pipeline includes biologics with novel mechanisms and advanced technology platforms such as gene therapy. The Baxalta Global Innovation and R&D Center is located in Cambridge, Massachusetts. Launched in 2015 following separation from Baxter International, Baxalta’s heritage in biopharmaceuticals spans decades. Baxalta’s therapies are available in more than 100 countries and it has advanced biological manufacturing operations across 12 facilities, including state-of-the-art recombinant production and plasma fractionation. Headquartered in Northern Illinois, Baxalta employs 16,000 employees worldwide.
Forward-Looking Statements
This release includes forward-looking statements concerning OBIZUR, including expectations with regard to its potential impact on patients, related regulatory actions and commercial launch plans. Such statements are made of the date that they were first issued and are based on current expectations, beliefs and assumptions of management. Forward-looking statements are subject to a number of risks and uncertainties, many of which involve factors or circumstances that are beyond Baxalta's control and which could cause actual results to differ materially from those in the forward-looking statements, including the following: satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; changes in laws and regulations; product quality, manufacturing or supply issues; patient safety issues; and other risks identified in Baxalta's Registration Statement on Form 10 and other Securities and Exchange Commission filings, all of which are available on Baxalta's website. Baxalta expressly disclaims any intent or obligation to update these forward-looking statements except as required by law.
REFERENCES
1. Kruse-Jarres R et al. Haemophilia 2015;21:162–170
2. Acquired Hemophilia: Revised Edition. World Federation of Hemophilia. 2012; No. 3: 1-5. Accessed on July 16, 2014. Available at: http://www1.wfh.org/publication/files/pdf-1186.pdf
3. Musial, J; Zdziarska, J. Acquired hemophilia A: an underdiagnosed, severe bleeding disorder. Department of Hematology, Jagiellonian University Medical College. 2nd Department of Medicine, Jagiellonian University Medical College. 2014. Accessed on July 16, 2014. Available at: http://pamw.pl/sites/default/files/PAMW%202014-04_Zdziarska.pdf
4. Franchini, M; Gandini, G; Paolantonio, T; Mariani, G. Acquired Hemophilia A; A Concise Review. American Journal of Hematology. 2005. No. 80: 55-63. Accessed on July 16, 2014. Available at: http://onlinelibrary.wiley.com/doi/10.1002/ajh.20390/pdf
5. Franchini, M; Mannucci, P. Acquired Hemophilia A: A 2013 Update. Department of Transfusion Medicine and Hematology, Carlo Poma Hospital, Mantova, Italy. 2013. Accessed on July 17, 2014. Available at: http://www.ncbi.nlm.nih.gov/pubmed/24008306
