Baxalta’s OBIZUR For Acquired Hemophilia A Receives Positive Opinion From European Committee For Medicinal Products For Human Use (CHMP)

• Positive opinion based on clinical data showing 100 percent of patients responded to treatment within 24 hours; European Commission approval anticipated later in 2015
• When approved, OBIZUR [Antihemophilic Factor (Recombinant), Porcine Sequence] will be the first recombinant porcine Factor VIII treatment approved in Europe, advancing care for acquired hemophilia A by enabling physicians to monitor treatment response by measuring FVIII activity levels
• OBIZUR is part of Baxalta’s comprehensive portfolio of inhibitor treatments for people living with bleeding disorders

DEERFIELD, 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 Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for the marketing authorization of OBIZUR [Antihemophilic Factor (Recombinant), Porcine Sequence].

“The marketing authorization anticipated later this year for OBIZUR will be an important milestone, offering patients with acquired hemophilia A in Europe a treatment option that transforms their care by allowing physicians to monitor treatment response”

Baxalta is seeking market authorization in Europe of OBIZUR for the treatment of bleeding episodes in adult patients with acquired hemophilia caused by antibodies to Factor VIII (FVIII), a very rare and potentially life-threatening acute bleeding disorder. Following this positive opinion, the European Commission is expected to make a decision on the application later this year. Upon approval in Europe, OBIZUR will be the first recombinant porcine FVIII treatment available for acquired hemophilia A, allowing physicians to monitor treatment response by measuring FVIII activity levels in addition to clinical assessments.

“The marketing authorization anticipated later this year for OBIZUR will be an important milestone, offering patients with acquired hemophilia A in Europe a treatment option that transforms their care by allowing physicians to monitor treatment response,” said John Orloff, M.D., head of Research & Development and chief scientific officer, Baxalta. “The positive support by the European regulators reflects the value this therapy can provide to patients, and supports Baxalta’s long-standing commitment to addressing unmet needs and reducing the global burden of bleeding disorders.”

The CHMP positive opinion is based on 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 adults with acquired hemophilia 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 least greater than 20 percent at 24 hours after the initial infusion. Resolution of the initial bleeding episode was observed in 24 out of 28 (86 percent) of the patients treated with OBIZUR. Common adverse reactions observed in greater than five percent of patients in the clinical trial were development of inhibitors to porcine FVIII.

OBIZUR is currently approved in the United States and is under regulatory review in Canada, Switzerland, Australia and Colombia. OBIZUR was granted orphan-drug designation by the European Commission based on the classification of acquired hemophilia A as a rare disease and the potential for the treatment to address an important unmet medical need.

About OBIZUR in Europe

OBIZUR [Antihemophilic 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 not indicated for the treatment of congenital hemophilia A or von Willebrand disease.

Important Risk Information for OBIZUR

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, 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 catheterisation 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.

Monitoring Laboratory Tests

Monitor Factor VIII activity and clinical condition 30 minutes after the first injection and 3 hours after administering OBIZUR.

Monitor Factor VIII activity immediately prior to and 30 minutes after subsequent doses and refer to the table for recommended target Factor VIII trough levels.

The one-stage clotting assay for Factor VIII is recommended as it has been used in determination of the potency of OBIZUR and the mean recovery rate.

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 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^1,2. In acquired hemophilia A, individuals typically experience subcutaneous, soft tissue, and post-surgical bleeding.^2,3,4 The comorbidities in this typically elderly population also pose a particular challenge to treat serious bleeding episodes.¹

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.		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
2.		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
3.		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
4.		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