Baxalta Gains EU Marketing Authorization To Provide OBIZUR To Adult Patients With Acquired Hemophilia A

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 the European Commission has granted Marketing Authorization for OBIZUR [Antihaemophilic Factor (Recombinant), Porcine Sequence].

OBIZUR is indicated for the treatment of bleeding episodes in adult patients with acquired haemophilia caused by antibodies to Factor VIII (FVIII), a very rare and potentially life-threatening acute bleeding disorder. OBIZUR is the first recombinant porcine sequence FVIII treatment available in Europe to treat acquired haemophilia A, and is designed to enable physicians to monitor treatment response by measuring FVIII activity levels in addition to clinical assessments.

“Gaining Marketing Authorization for this first recombinant porcine option for acquired haemophilia in Europe reflects Baxalta’s commitment to improving patient lives,” said Brian Goff, executive vice president and president, Haematology, Baxalta. “We continue to build a broad, global portfolio of treatments that aim to reduce the burden of bleeding disorders for patients as we pursue a world without bleeds.”

The approval is supported by the positive results of a global, prospective, controlled, multi-centre Phase II/III open-label clinical trial that examined the efficacy and safety of OBIZUR in the treatment of serious bleeding episodes in adults with acquired haemophilia A (29 patients evaluated for safety, 28 evaluated for efficacy). All patients treated with OBIZUR (28/28) showed a positive response (bleeding stopped or reduced) and clinical improvement, with FVIII activity levels at 20 percent or higher 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 factor VIII was the only adverse reaction reported in more than five percent of patients.¹

Acquired haemophilia A is a rare haemorrhagic disease in which autoantibodies against coagulation factor VIII, called FVIII-neutralizing antibodies (inhibitors), weaken the inherent coagulation system. As the inhibitors developed in AHA are autoantibodies, the disease may be caused by and is often associated with autoimmune disease. Acute haemorrhage associated with AHA may be fatal, however AHA is often unrecognized or misdiagnosed and should thus be considered in the differential diagnosis particularly in postpartum women and the elderly with bleeding tendency or prolonged activated partial thromboplastin time.²

About OBIZUR in Europe

OBIZUR [Antihaemophilic Factor (Recombinant), Porcine Sequence] is indicated for the treatment of bleeding episodes in adults with acquired haemophilia caused by antibodies to Factor VIII.

OBIZUR is now approved in the United States, Canada and Europe, and is under regulatory review in Switzerland, Australia and Colombia.

Important Risk Information for OBIZUR

This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions.

CONTRAINDICATIONS

OBIZUR should not be used in patients with known anaphylactic reactions to the active substance, hamster protein, or to any of the excipients.

WARNINGS & PRECAUTIONS

Hypersensitivity

Allergic type hypersensitivity reactions are possible with OBIZUR. The product contains trace amounts of hamster proteins.

If symptoms of hypersensitivity occur, patients should be advised to discontinue use of the medicinal product immediately and contact their physician. Patients should be informed of the early signs of hypersensitivity reactions including hives, generalised urticaria, and tightness of the chest, wheezing, hypotension, and anaphylaxis.

In case of shock, standard medical treatment for shock should be implemented.

Development of inhibitory antibodies

Inhibitory antibodies against porcine Factor VIII (measured using a modification of the Nijmegen variation of the Bethesda assay) were detected before and after exposure to OBIZUR. Inhibitor titres of up to 29 Bethesda units were recorded at baseline yet subjects responded positively to OBIZUR. It is recommended that treatment should be based on clinical judgement and not based on detection of inhibitory antibodies by the Bethesda assay.

There is a lack of clinical information on the development of inhibitory antibodies to OBIZUR following repeated administration. Therefore, OBIZUR must only be administered when considered clinically necessary. Extensive cutaneous purpura do not necessarily require treatment.

OBIZUR is produced by recombinant DNA technology in baby hamster kidney cells. Antibodies to baby hamster kidney cell protein were not detected in subjects either before or after exposure to OBIZUR.

High and sustained Factor VIII activity in blood may predispose to thromboembolic events. Those with pre-existing cardiovascular disease and the elderly are at particular risk.

If venous catheterization is required, the risk of catheter-related complications such as catheter site thrombosis should be considered.

Factor VIII activity determined by the chromogenic assay is generally lower than Factor VIII activity determined by the one-stage clotting assay. Measurement of Factor VIII activity must always be carried out using the same assay methodology on any one patient. The one-stage assay is recommended because it has been used in determination of the potency and the mean recovery rate of OBIZUR.

Sodium content

Each vial contains 4.4 mg (198 mM) sodium per ml of reconstituted solution. To be taken into consideration by patients on a controlled sodium diet.

ADVERSE REACTIONS

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

About Acquired Haemophilia A

Acquired haemophilia A is a rare, potentially life-threatening bleeding disorder, which, unlike congenital haemophilia, typically affects older adults and occurs in both males and females.^3,4 In acquired haemophilia A, individuals typically experience subcutaneous, soft tissue, and post-surgical bleeding.^4,5,6 The comorbidities in this typically elderly population also pose a particular challenge to treat serious bleeding episodes.³

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 haematology, 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 Centre 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. Sakurai, Y., Takeda, T., ‘Acquired Hemophilia A: A Frequently Overlooked Autoimmune Hemorrhagic Disorder’, Journal of Immunology Research, Volume 2014 (2014), Article ID 320674, 10 pages. Available at: http://www.hindawi.com/journals/jir/2014/320674/

3. 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

4. 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

5. 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

6. 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