FDA Gives Nod to Baxalta’s Rare Bleeding Disorder Drug Vonvendi

Baxalta Receives FDA Approval for VONVENDI, the First and Only Recombinant Treatment for Adults Affected by Von Willebrand Disease

- von Willebrand disease (VWD) is a genetic disorder that results in impaired clotting with limited treatment options today
- VONVENDI [von Willebrand factor (Recombinant)] represents the most significant therapeutic advancement for the treatment of adults with VWD in more than a decade, offering a new approach to treat the disease
- In clinical studies, VONVENDI demonstrated 100 percent treatment success in resolving bleeding episodes with a median of only two infusions to treat severe bleeds

BANNOCKBURN, Ill.--(BUSINESS WIRE)--

Baxalta Incorporated (BXLT), a global biopharmaceutical leader dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, announced today that the U.S. Food and Drug Administration (FDA) has approved VONVENDI [von Willebrand factor (Recombinant)]. VONVENDI is the first and only recombinant treatment for adults living with von Willebrand Disease (VWD).

VONVENDI is an innovative recombinant protein treatment that includes a physiologic distribution of proteins called multimers, including ultra-large multimers (ULMs), with large multimers being the most active form of the protein supporting clot formation. The treatment is also the first in the United States that contains only trace amounts of Factor VIII (FVIII), offering the flexibility to administer FVIII only when needed. This attribute allows for tailored treatment for patients who may not require additional FVIII.

“With no major therapeutic innovation in more than a decade, VONVENDI offers patients an important new option for VWD with a clinical profile that can help them manage this challenging chronic disease,” said Joan Gill, M.D., of the Blood Center and the Medical College of Wisconsin and lead author of the VONVENDI clinical study.

VWD is the most common inherited bleeding disorder worldwide, affecting up to one in 100 people; the rarest and often most severe form affects one in 1,000,000 people worldwide. This genetic disorder causes alterations or deficits in von Willebrand factor (VWF), resulting in impaired clotting, and affects women and men equally. The disease can manifest through a variety of bleeding events, including mucosal bleeds, gastrointestinal bleeds or menorrhagia. Patients often live with the disease for years without a proper diagnosis; even with a confirmed diagnosis, there are limited treatment options available.

“With its unique feature of ultra-large multimers and the clinical profile seen in our pivotal study, VONVENDI is an important new option with the potential to redefine treatment for adults with von Willebrand disease,” said Brian Goff, executive vice president and president, Hematology, Baxalta. “VONVENDI’s approval represents our commitment to advancing care as we continue to build a strong and differentiated portfolio that can help address challenging bleeding disorders for patients around the world.”

VONVENDI has been approved for on-demand treatment and control of bleeding episodes in adults with von Willebrand disease. The FDA approval was based on positive results from a Phase III multicenter, open-label clinical trial that assessed the safety, efficacy and pharmacokinetics of VONVENDI with and without recombinant FVIII. In the pivotal study, all participants (100 percent) reported successful treatment of bleeding episodes, with 96.9 percent of treated bleeds (N=192 bleeds in 22 patients) achieving an “excellent” efficacy rating and 3.1 percent achieving a “good” efficacy rating. Most bleeds (81.8 percent) were resolved with a single infusion, and the treatment showed a mean half-life of 21.9 hours (± 8.36).^1,2

No thrombotic events or severe product-related adverse events were observed during the clinical trial, nor were there treatment-related binding or neutralizing antibodies against VWF or neutralizing antibodies against FVIII. The most common adverse reaction observed in greater than two percent of patients in clinical trials was generalized pruritus.

Baxalta is building a robust clinical development program to optimize patient access to VONVENDI worldwide; a series of clinical programs are planned to evaluate its use for prophylaxis, surgical and pediatric indications. VONVENDI is expected to be broadly available in the United States in late 2016. Baxalta expects to file for regulatory approvals in Europe in 2017 and in other markets around the world.

About VONVENDI [von Willebrand factor (Recombinant)]

VONVENDI [von Willebrand factor (Recombinant)] is a recombinant von Willebrand factor indicated for on-demand treatment and control of bleeding episodes in adults (age 18 and older) diagnosed with von Willebrand disease.

Detailed Important Risk Information for VONVENDI

CONTRAINDICATIONS

VONVENDI is contraindicated in patients who have had life-threatening hypersensitivity reactions to VONVENDI or constituents of the product (tri-sodium citrate-dihydrate, glycine, mannitol, trehalose-dihydrate, polysorbate 80, and hamster or mouse proteins).

WARNINGS AND PRECAUTIONS

Embolism and Thrombosis

Thromboembolic reactions, including disseminated intravascular coagulation (DIC), venous thrombosis, pulmonary embolism, myocardial infarction, and stroke, can occur, particularly in patients with known risk factors for thrombosis. Monitor for early signs and symptoms of thrombosis such as pain, swelling, discoloration, dyspnea, cough, hemoptysis, and syncope.

In patients requiring frequent doses of VONVENDI with recombinant factor VIII, monitor plasma levels for FVIII:C activity because an excessive rise in factor VIII levels can increase the risk of thromboembolic complications.

Hypersensitivity Reactions

Hypersensitivity reactions, including anaphylaxis, may occur. Symptoms can include anaphylactic shock, generalized urticaria, angioedema, chest tightness, hypotension, shock, lethargy, nausea, vomiting, paresthesia, pruritus, restlessness, wheezing and/or acute respiratory distress. If signs and symptoms of severe allergic reactions occur, immediately discontinue administration of VONVENDI and provide appropriate supportive care.

Neutralizing Antibodies

Neutralizing antibodies (inhibitors) to von Willebrand factor and/or factor VIII can occur. If the expected plasma levels of VWF activity (VWF:RCo) are not attained, perform an appropriate assay to determine if anti-VWF or anti-FVIII inhibitors are present. Consider other therapeutic options and direct the patient to a physician with experience in the care of either von Willebrand disease or hemophilia A. In patients with high levels of inhibitors to VWF or factor VIII, VONVENDI therapy may not be effective and infusion of this protein may lead to severe hypersensitivity reactions. Since inhibitor antibodies can occur concomitantly with anaphylactic reactions, evaluate patients experiencing an anaphylactic reaction for the presence of inhibitors.

ADVERSE REACTIONS

The most common adverse reaction observed in =2% of subjects in clinical trials (n=66) was generalized pruritus.

Please see VONVENDI full Prescribing Information: http://www.baxalta.com/assets/documents/VONVENDI_PI.pdf.

About Baxalta

Baxalta Incorporated (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 VONVENDI and Baxalta’s R&D pipeline, including expectations with regard to clinical trials, future regulatory actions, commercial launch plans and potential impact on patients. 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: demand for and market acceptance of risks for new and existing products; clinical trial results; 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; the impact of competitive products and pricing; and other risks identified in Baxalta’s filings with the Securities and Exchange Commission, 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. VONVENDI product information. http://www.baxalta.com/assets/documents/VONVENDI_PI.pdf.
2. Gill JC, et al. Hemostatic efficacy, safety and pharmacokinetics of a recombinant von Willebrand factor in severe von Willebrand disease. Blood. 2015.

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