Client Presentation

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Simple • Ready-to-use 10% liquid IVIg • Sucrose-free • Latex-free • Complimentary reimbursement hotline

Ready-to-Use Liquid Privigen™ at a Glance Low Annual Rate of Serious Bacterial Infections* Per Subject Year† (n=80 Subjects)

1.0 0.9 0.8 0.7 0.6 0.5 0.4 0.3 0.2 0.1 0.0

Multiple Processing and Viral Inactivation/Removal* Steps

Annual Percentage of Sick Days vs Days Able to Perform Normal Activities

8.65 Days

2%

Sick Days 356.35 Days

• Depth filtration (CH9 Z+)*

• Cold ethanol precipitation

• Anion exchange chromatography

• Nanofiltration (to 20 nm)*

• Clarifying depth filtration

• Ultrafiltration

• Low pH incubation (inactivation under acidic conditions)*

• Octanoic acid fractionation

Purity

98%

Specification

Typical Value

Excipient

Concentration

IgG

≥98.0%

>99.2%†

L-proline

250 mmol/L (210-290 mmol/L)

Days Able to Perform Normal Activities

0.08

Composition

Protein IgG1

67.8% (61.7%-72.8%)

IgG2

28.7% (23.8%-35.3%)

Sodium

≤1 mmol/L

IgG3

2.3% (1.8%-2.9%)

Carbohydrate Stabilizers (Sucrose, Maltose, etc)

None

IgG4

1.2% (0.5%-2.8%)

IgA

≤25 mcg/mL

16-37 mcg/mL

Physicochemical and Pharmacokinetic Properties

* Results from case report forms

Aggregates

≤2.0%

<0.1% †

Parameter

Value

† Upper 1-sided 99% confidence interval: 0.203

Dimers

≤12.0%

4.4% at release, may increase to 8% during shelf-life†

Osmolality

320 mOsmol/kg (240-440 mOsmol/kg)

pH

4.8 (4.6-5.0)

Fragments

≤3% at release ≤6% at end of shelf-life

<1.9% at release, may increase to ≈ 5% during shelf-life†

Viscosity

~3 mPas at room temperature

Median half-life

36.6 days

Confirmed aSBIs‡

‡ Acute serious bacterial infections (aSBIs) defined as pneumonia, bacterial meningitis, bacteremia/septicemia, osteomyelitis/septic arthritis, and visceral abscess

Innovative • First and only proline-stabilized liquid Ig therapy • Only approved 10% liquid with 24-month room temperature storage • Low IgA content (≤25 mcg/mL)

Mean of 29 batches; Data on file. CSL Behring

Titers of Antibodies in Assays of Privigen™

What is proline?

Room Temperature Storage (months) 24

Privigen™ Gammagard Liquid*

9 6

Gamunex† 2

4

6

8 10 12 14 16 18 20 22 24

*Gammagard is a registered trademark of Baxter International, Inc.

Proline is one of the 20 naturally occurring amino acids that form human proteins. It can be synthesized by the human body or obtained from dietary sources. CSL Behring chose proline as the stabilizer for Privigen™ after extensive testing, analysis, and comparison to other amino acids, such as glycine.

Antibody

Range

Diphtheria antitoxin

3.8-7.3 IU/mL

Anti-streptolysin O

1310-2010 IU/mL

Anti-tetanus toxoid

26.9-52.1 IU/mL

Anti-pneumococcal capsular polysaccharide

595-1065 mcg/mL

Anti-hepatitis B surface antigen

3.0-10.1 IU/mL

Anti-poliomyelitis virus type 1

19-67 IU/mL

Anti-Haemophylus influenzae B

26.4-45.0 IU/mL

Anti-cytomegalovirus

51.2-116.8 IU/mL

†Gamunex is a registered trademark of Talecris Biotherapeutics, Inc.

Spec Sheet

7.1 Mechanism in Immunodeficiency

Figure 19. Blockade of Fcγ Receptors

is used by natural killer cells (NK cells) for removal of virus-infected cells or by eosinophils for killing of parasites.

In patients with primary and secondary immunodeficiencies, Privigen™ is used to provide the missing protective antibodies (replacement therapy). Privigen™ is manufactured from the pooled plasma of up to 60,000 blood donors and therefore includes a broad variety of antibody specificities against bacteria, viruses, other pathogens, and toxins to which the donor population has been exposed.92 Moreover, the antibodies in Privigen™ are structurally and functionally intact and their effector functions are fully operative. Binding to Fcγ receptors and activation of complement are important for the destruction of pathogens through phagocytosis or complement-mediated lysis and for the clearance of immune complexes. Antibody-dependent cellular cytotoxicity

Beyond its protective effect against infections, IgG has an important role in the regulation of the function of the whole immune system. Patients with primary immune deficiency often develop autoimmune disease. For example, patients with common variable immunodeficiency (CVID) often have immune thrombocytopenic purpura (ITP) or autoimmune hemolytic anemia (AIHA). Intravenous immunoglobulin substitution therapy is believed to reduce the frequency of these autoimmune manifestations in PI.93

7.2 Mechanisms in Immunomodulation

Blocking of activating Fcγ receptors by infused IgG molecules may also inhibit antibody-dependent cellular cytotoxicity, as suggested for the protective effect of IVIg against demyelination in inflammatory neurologic diseases.96 Studies in a murine model, in which ITP was induced by an anti-platelet antibody, suggested another mechanism for

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b. Activated macrophages phagocytose and destroy antibody-coated platelets.

c. A possible mechanism of action of IVIg in ITP is the blockade of Fcγ receptors, inhibiting the destruction of platelets coated with pathogenic autoantibodies.

d. Another hypothesis is that IVIg treatment decreases the expression of activating Fcγ receptors and/or increases the expression of the inhibiting FcRIIb. The macrophage response is thus shifted from activation to inhibition, and antibody-coated platelets are not phagocytosed. So far, this mechanism has only been demonstrated in mouse models of ITP.

down-regulation of phagocytosis. Platelet consumption was prevented by administration of IVIg or its Fc fragments and the effect was dependent on the surface expression of FcγRIIb on splenic macrophages. 97 The low affinity FcγRIIb (CD32) is an inhibitory receptor that blocks the clearance of platelets (Figure 19). 7.22 Modulation of Complement Activation and Other Anti-inflammatory Effects With its potent anti-inflammatory activity, IVIg has rapid effects on acute inflammation as has been shown in Kawasaki disease. 98,99 A major mechanism mediating IVIg anti-inflammatory effect is complement scavenging. Activated complement components, particularly C3b can bind to IgG. The infusion of a high dose of IVIg offers a multitude of binding sites that can re-direct the complement attack from the target organ (tissue recognized by pathogenic autoantibodies) to the IgG in the liquid phase. This effect has been shown in an animal model100 and in vitro and in vivo studies of patients with dermatomyositis.101,102

MOA

7.21 Blockade or Modulation of Fcγ Receptors The primary mechanism by which Privigen™ prevents the uncontrolled phagocytosis of autoantibody-coated platelets in ITP is considered to be a blockade of Fcγ receptors. This mechanism, which is supported by several lines of evidence, is illustrated in Figure 19. IVIg treatment has been shown to inhibit the clearance of RhD positive autologous erythrocytes coated with anti-RhD antibodies.94 Whether one of the three activating Fcγ receptors (FcγRI [CD64], FcγRIIa [CD32], FcγRIII [CD16]) is preferably inhibited by IgG, and to which degree monomeric, dimeric or complexed IgG (formed during or after infusion) are involved in IVIg-induced inhibition is still a matter of debate.89,95

a. In ITP, platelets are coated with pathogenic autoantibodies. These attach to splenic macrophages via activating Fcγ receptors.

Activated Fcγ receptor

Inhibitory Fcγ receptor

Autoantibody

IVIg

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Monograph

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