Baxalta Announces Expanded CMS Coverage For Medicare PI Patients To Receive At-Home Treatment With HYQVIA

• U.S. PI patients on Medicare now covered to self-administer HYQVIA [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] at home
• Baxalta continues to expand access to HYQVIA as the only once-monthly subcutaneous treatment to reduce the burden of primary immunodeficiency for patients worldwide

DEERFIELD, Ill.--(BUSINESS WIRE)--Baxalta Incorporated, a global biopharmaceutical leader dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, today announced that the Centers for Medicare and Medicaid Services (CMS) have expanded coverage to include in-home use of HYQVIA [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase], a treatment for adults with primary immunodeficiency (PI).

“This coverage expansion allows for Medicare patients and their physicians to select the site of care for HYQVIA that best fits their treatment needs and lifestyle, which may be valuable for many of the more than 10,000 PI patients in the Medicare system.”

PI is the name for a group of more than 200 diseases wherein part of the person’s immune system is missing or not functioning correctly.¹ People with PI may be more likely to get infections, take longer to recover from infections and have recurring infections. HYQVIA offers a demonstrated safety and efficacy profile while delivering a full dose of treatment via one needle, one infusion site, once per month.

Following the U.S. Food and Drug Administration (FDA)’s approval of HYQVIA in 2014, CMS covered both provider facility and in-office treatment with HYQVIA. This new decision expands its provisions to include durable medical equipment (DME) coverage of the infusion pump required to administer HYQVIA, better enabling an at-home, self-administration option for patients.

According to treatment preferences stated by PI patients who participated the clinical trial for HYQVIA, the majority of patients expressed a desire for the ability to administer infusions at home.²

“It is important that patients have the option to be treated at the site that works best for their personal needs, and many people with PI have voiced a preference for in-home care,” said Dr. Amy Darter of the Oklahoma Institute of Allergy & Asthma. “This decision to expand coverage is a critical step forward in meeting their needs and equipping them to better manage their disease.”

“Today’s decision from CMS reinforces the value of HYQVIA and will help expand access to even more people who can benefit from the flexibility of self-administering HYQVIA in their own homes,” said Jacopo Leonardi, executive vice president and president, Immunology, Baxalta. “This coverage expansion allows for Medicare patients and their physicians to select the site of care for HYQVIA that best fits their treatment needs and lifestyle, which may be valuable for many of the more than 10,000 PI patients in the Medicare system.”

For more information, review the letter at https://www.medicarenhic.com/dme/whatsnew.aspx or learn more about HYQVIA at www.HYQVIA.com.

About HYQVIA [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase]

Indication and Usage

HYQVIA is an immune globulin with a recombinant human hyaluronidase indicated for the treatment of Primary Immunodeficiency (PI) in adults. This includes, but is not limited to, common variable immunodeficiency (CVID), X-linked agammaglobulinemia, congenital agammaglobulinemia, Wiskott-Aldrich syndrome, and severe combined immunodeficiencies.

Limitation of Use:

Safety and efficacy of chronic use of recombinant human hyaluronidase in HYQVIA have not been established in conditions other than PI.

Detailed Important Risk Information

BOXED WARNING: THROMBOSIS

Thrombosis may occur with immune globulin products, including HYQVIA. Risk factors may include advanced age, prolonged immobilization, hypercoagulable conditions, history of venous or arterial thrombosis, use of estrogens, indwelling vascular catheters, hyperviscosity, and cardiovascular risk factors. Thrombosis may occur in the absence of known risk factors. For patients at risk of thrombosis, administer HYQVIA at the minimum dose and infusion rate practicable. Ensure adequate hydration in patients before administration. Monitor for signs and symptoms of thrombosis and assess blood viscosity in patients at risk of hyperviscosity.

CONTRAINDICATIONS

HYQVIA is contraindicated: in patients who have a history of anaphylactic or severe systemic hypersensitivity reactions to the administration of Human Immune Globulin (IgG); in IgA-deficient patients with antibodies to IgA and a history of hypersensitivity; and in patients with known systemic hypersensitivity to hyaluronidase or Recombinant Human Hyaluronidase of HYQVIA.

WARNINGS and PRECAUTIONS

Hypersensitivity: Severe hypersensitivity reactions may occur, even in patients who have tolerated previous treatment with IgG. IgA-deficient patients with antibodies to IgA are at greater risk of developing potentially severe hypersensitivity and anaphylactic reactions.

Thrombosis: Thrombosis may occur following treatment with immune globulin products, including HYQVIA. Risk factors may include advanced age, prolonged immobilization, hypercoagulable conditions, history of venous or arterial thrombosis, use of estrogens, indwelling central vascular catheters, hyperviscosity, and cardiovascular risk factors. Thrombosis may occur in the absence of known risk factors.

Immunogenicity of Recombinant Human Hyaluronidase (PH20):

Non-neutralizing antibodies to the recombinant human hyaluronidase component can develop. The potential exists for such antibodies to cross-react with endogenous PH20, which is known to be expressed in adult male testes, epididymis, and sperm. The clinical significance of these antibodies or whether they interfere with fertilization in humans is unknown.

Aseptic Meningitis Syndrome (AMS): AMS has been reported to occur with IgG treatment administered intravenously and subcutaneously. Discontinuation of IgG treatment has resulted in remission of AMS within several days without sequelae.

Hemolysis: Acute intravascular hemolysis has been reported following intravenously administered IgG products, including Immune Globulin Infusion 10% (Human) administered intravenously, and delayed hemolytic anemia can develop due to enhanced RBC sequestration. IgG products, including HYQVIA, contain blood group antibodies which may cause a positive direct antiglobulin reaction and hemolysis.

Renal Dysfunction/Failure: Acute renal dysfunction/failure, acute tubular necrosis, proximal tubular nephropathy, osmotic nephrosis, and death may occur upon use of IgG products administered intravenously, especially those containing sucrose. HYQVIA does not contain sucrose. Ensure that patients are not volume depleted prior to the initiation of infusion of HYQVIA. Monitor renal function and urine output and consider lower, more frequent dosing in patients who are at risk of developing renal dysfunction because of pre-existing renal insufficiency or predisposition to acute renal failure.

Spread of Localized Infection: Do not infuse HYQVIA into or around an infected or acutely inflamed area due to potential risk of spreading a localized infection.

Transfusion-Related Acute Lung Injury (TRALI): Non-cardiogenic pulmonary edema has been reported in patients following treatment with intravenously administered IgG products, including Immune Globulin Infusion 10% (Human). TRALI is characterized by severe respiratory distress, pulmonary edema, hypoxemia, normal left ventricular function, and fever.

Transmittable Infectious agents: Because the Immune Globulin Infusion 10% (Human) of HYQVIA is made from human plasma, it may carry a risk of transmitting infectious agents, e.g., viruses and other pathogens, and theoretically, the Creutzfeldt-Jakob disease (CJD) agent. No cases of viral transmission or CJD have been associated with HYQVIA.

Interference with Laboratory Tests: False positive serological test results, with the potential for misleading interpretation, may result from the transitory rise of the various passively transferred antibodies in the patient’s blood after infusion of IgG. Passive transmission of antibodies to erythrocyte antigens (e.g., A, B, and D) may cause a positive direct or indirect antiglobulin (Coombs’) test.

ADVERSE REACTIONS

The most common adverse reactions observed in > 5% of patients in the clinical trials were: local adverse reactions (52%), headache (21%), antibody formation against recombinant human hyaluronidase (18%), fatigue (11%), nausea (7%), pyrexia (7%), and vomiting (7%). No serious adverse reactions occurred during the HYQVIA clinical trials.

Please see the Full Prescribing Information, including Boxed Warning for HYQVIA at: http://www.baxalta.com/assets/documents/HYQVIA_PI.pdf.

About Primary Immunodeficiency

Primary immunodeficiencies (PI) are a group of more than 200 disorders in which part of the body’s immune system is missing or does not function properly.² Normally, the immune system protects the body from pathogenic microorganisms like bacteria, viruses, and fungi, which can cause infectious diseases. When any part of a person’s immune system is absent or dysfunctional, they are susceptible to infections and may take longer to recover from infections. When a defect in the immune system is inherited, it is called primary immune deficiency. It is estimated that as many as six million children and adults are affected by PI worldwide.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 HYQVIA, including expectations with regard to its potential impact on patients. Such statements are made as 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. Bousfiha AA et al. Primary immunodeficiency diseases worldwide: more common than generally thought. J Clin Immunol. 2013 Jan;33(1):1-7.

2. Wasserman R. et al. Recombinant human hyaluronidase-facilitated subcutaneous infusion of human immunoglobulins for primary immunodeficiency. J Allergy Clinical Immunology. 2012;130(4): 951-957.