Kedrion Launches FDA-Approved Gammaked in U.S. Market

The U.S. Food and Drug Administration has approved Kedrion Biopharma’s Gammaked, a 10 percent liquid, ready-to-use sterile solution of human immune globulin, for the U.S. market. Gammaked is approved for intravenous administration for primary immunodeficiency, idiopathic thrombocytopenic purpura and chronic inflammatory demyelinating polyneuropathy, and for subcutaneous administration to treat primary immunodeficiency. It is supplied in 1-, 2.5-, 5-, 10- and 20-gram single-use bottles. Kedrion has entered into an agreement with Grifols SA to manufacture Gammaked for the next seven years. And, as part of its ongoing expansion in the U.S., Kedrion began distribution of Gammaked on August 2 through designated channel partners.

New Medicare IVIG Access Bills Introduced

Two new intravenous immune globulin (IVIG) access bills, one in the House and one in the Senate, have been introduced. H.R. 1845, introduced by Representatives Kevin Brady (R-Texas) and Doris Matsui (D-Calif.), and S. 960, introduced by Senators John Kerry (D-Mass.) and Lamar Alexander (R-Tenn.), both titled Medicare IVIG Access Act, provide for a study of issues relating to access to IVIG for Medicare beneficiaries in all care settings and a demonstration project to examine the benefits of providing coverage and payment for items and services necessary to administer IVIG in the home for patients with primary immunodeficiency diseases (PIDDs). Current law pays for IVIG but prohibits Medicare from reimbursing for nursing services and equipment necessary to infuse a patient in the home setting.

According to an Immune Deficiency Foundation patient survey, when compared to private insurance, Medicare patients share a disproportionate burden of negative consequences as a result of the changes in Medicare IVIG reimbursement policies. Since January 2005, 32 percent of Medicare patients reported they have been forced to change their preferred IVIG treatment location. Many patients now must receive IVIG in hospitals, which is not the ideal location for PIDD patients who are especially susceptible to opportunistic infections.

FDA Extends Shelf Life of Hizentra to
30 Months

The U.S. Food and Drug Administration has approved a supplemental Biologics License Application to extend the shelf life of Hizentra, Immune Globulin Subcutaneous (Human), 20% Liquid, from 24 months to 30 months. Stabilized with L-proline, a naturally occurring amino acid, Hizentra can be stored at room temperature (up to 25 degrees Celsius or 77 degrees Fahrenheit) for up to 30 months, and because no refrigeration is necessary, it can be ready to use without warning, offering patients and physicians convenience and portability.

Hizentra is the first and only 20 percent subcutaneous immune globulin approved in the U.S. It is indicated for the treatment of primary humoral immunodeficiency, a group of disorders that result from a dysfunctional immune system that prevent patients from fighting off infections caused by common germs.

Grifols Expands PatientCare Program

Grifols Inc. has expanded its PatientCare Program to offer intravenous immune globulin (IVIG) to the primary immune deficiency (PIDD) community. The program will provide Flebogamma 5% DIF (Immune Globulin Intravenous [Human]) or Flebogamma 10% DIF (Immune Globulin Intravenous [Human]) at no cost to patients with PIDD when insurance coverage is not available. The PatientCare Program consists of Grifols Assurance for Patients (GAP), which provides therapy for users of Grifols’ products during a lapse in insurance coverage, and Grifols Patient Assistance (GPA), which provides therapy for individuals in need of temporary assistance.

The GAP program is open to anyone diagnosed with PIDD who has used a Grifols’ product for the three months prior to lapse in coverage. The GPA program is open to anyone diagnosed with PIDD without insurance who is in need of temporary assistance, regardless of whether they have used a Grifols therapy. However, applicants of the GPA program must meet financial eligibility requirements, and both programs require certain insurance ineligibility. To take advantage of the program, patients must complete eligibility forms, which can be downloaded at www.grifolspatientcare.com, and submit them to a Grifols PatientCare representative for verification. Neither program is an emergency program, and if a lapse in coverage is anticipated, the eligibility forms should be submitted ahead of time.

Grifols also operates an Emergency Supply System for physicians seeking to obtain IVIG to treat a specific patient under emergency circumstances. Access to this emergency system does not require the requesting physician to be a current customer of Grifols, and there are no prerequisites other than an emergent need for treatment. For more information about the Emergency Supply System, contact (888) GRIFOLS.

Study Helps to Explain
IG Distribution in the Body

CSL Behring has developed a pharmacokinetic (PK) model that shows how the body absorbs, distributes, metabolizes and eliminates immunoglobulin (IG) following subcutaneous (SC) administration. The PK model provides a new means of simulating the mechanism by which SCIG is transported after it is injected into the subcutaneous tissue, which could affect the volume and frequency of IG dosing for primary immunodeficiency (PI) patients.

Currently, little is understood about the clinical implications of SC versus intravenous (IV) dosing of IG in PI patients, or about where SCIG travels within the body after it is administered and how long it remains there. This new model, which describes a complex system of continuous interactions between extravascular (tissue) and intravascular (blood) compartments that help determine the location and level of SCIG in different parts of the body after injection, shows that fluctuations in serum IgG after IVIG dosing were lower in the first half of the 30-day dosing cycle and higher in the second half of the cycle than would be anticipated. This, then, supports the theory that IgG distribution from the intravascular to the extravascular compartment occurs early in the dosing cycle, with the reverse occurring later in the dosing cycle.

“Clearly, a need exists to better understand the highly complex pharmacokinetic interactions that take place after an infusion of IgG, especially SCIG, in areas of the body that are not traditionally monitored by clinicians,” said Stephen Jolles, MD, University Hospital of Wales, Cardiff, U.K. “This new PK model represents a major step toward filling this need, especially for clinicians who measure serum IgG as a means of determining overall levels of IgG, and provides valuable insight into movement of IgG around the body with implications for improving PI patient dosing and treatment.”

Florida Adds SCID to Newborn Screening Panel

On January 28, Florida voted to add severe combined immunodeficiency (SCID) to the state’s newborn screening panel, a uniform set of newborn screening tests. Florida joins five states and one territory in its decision to add SCID to its panel: California, Louisiana, Massachusetts, New York, Wisconsin and Puerto Rico. In addition, Texas has a limited pilot SCID screening panel in place, where it tested approximately 20,000 infants in 2010.

In the United States, newborn screening began more than 40 years ago, when states and territories mandated newborn screening of all infants born within their jurisdiction for certain disorders that may not otherwise be detected before developmental disability or death occurs. Newborns with these disorders typically appear normal at birth, so when detected at birth, these diseases can be medically treated to allow most affected newborns to develop normally.

Seven other states also have voted to recommend the addition of SCID to their newborn screening panels, but screening has not yet begun. These include Colorado, Delaware, Iowa, Michigan, Minnesota, North Carolina and Rhode Island. In addition, proposals to add SCID have been made in Connecticut, Nebraska, Ohio and Pennsylvania.

Gamunex-C Approved for Subcutaneous Administration

The U.S. Food and Drug Administration (FDA) has approved Talecris Biotherapeutics’ Gamunex-C (immune globulin injection [human] 10% caprylate/chromatography purified) for subcutaneous administration in the treatment of primary immunodeficiency (PIDD). Gamunex-C can be administered subcutaneously and intravenously, whereas Talecris' previously FDA-approved Gamunex can be administered intravenously only. Intravenous (IV) delivery for both products is FDA-approved to treat PIDD, chronic inflammatory demyelinating polyneuropathy (CIDP) and idiopathic thrombocytopenic purpura (ITP). Gamunex-C has labeling and packaging information that describes both IV and subcutaneous routes of administration. Gamunex has labeling and packaging information that describes only IV administration.

“The FDA approval of Gamunex-C is important because it provides another option for patients with primary immunodeficiency and their healthcare professionals when they are considering the various treatment modalities,” says Fred Modell, cofounder of the Jeffrey Modell Foundation. “We consider it significant for patients to have multiple modes of delivery so they can select the option that best suits their individual needs.”

FDA Approves Grifols' Flebogamma DIF 10% IVIG

Grifols has obtained U.S. Food and Drug Administration approval for its next generation of intravenous immunoglobulin (IVIG) 10% concentration, under the name Flebogamma 10% DIF. Flebogamma DIF (double inactivation and filtered) is a polyvalent IVIG that incorporates two specific viral inactivation methods and the additional safety step of nanofiltration at 20 nanometers. These processes produce higher yields of the product to maximize the amount of life-saving medicine that can be produced from each plasma donation.

With this approval, Grifols is the first company in the U.S. to offer patients and clinicians two concentrations of liquid IVIG (5% and 10%). The company plans to launch Flebogamma 10% DIF toward the end of 2010.

Octagam Withdrawal Issued for All Lots

Octapharma USA has initiated a voluntary withdrawal of all lots of Octagam (immune globulin intravenous [human] 5% liquid preparation) from the U.S. marketplace due to an unusually high number of thromboembolic events that have been associated with people being administered the drug. This follows an initial announcement in August of a voluntary withdrawal of selected lots of Octagam 5%, which reported at least nine events where blood clots dislodged and traveled through the body, causing injury and pain to patients.

According to Octapharma USA, while the company has not received any reports of thromboembolic events since its initial voluntary market withdrawal, the Food and Drug Administration and Octapharma agree that until a root cause analysis of the previously reported thromboembolic events can be determined, the most prudent course of action is to suspend further administration of Octagam 5%.

The company requests that customers quarantine all lots of Octagam 5% and then contact the Octapharma customer service department at (201) 604-1141 to return the product.

This withdrawal is for Octagam only. Octapharma's Albumin (Human) and Wilate, Von Willebrand Factor/Coagulation Factor VIII Complex (Human), are unaffected and are readily available in all sizes for purchase.

Grifols Buys Talecris for $4 Billion

Grifols, the global healthcare group based in Barcelona that produces blood plasma treatments, has agreed to acquire Talecris’ plasma-based sector for $4 billion, including debt. The company says it expects to save 230 million euros ($297 million) in operating costs in plasma collection, manufacturing, marketing and research, and that the combined company would have about $2.8 billion in yearly revenues, mostly from North America.

Victor Grifols, chief executive of Grifols, praised Talecris’ “strong clinical research capability and new research into recombinant therapies,” adding that the deal would expand Grifols’ product line, geographic reach and manufacturing scale. This buyout will reduce the number of companies in this U.S. market from five to four.