Recent Highlights
CSL continues to drive innovation in its therapeutic categories, including plasma-protein replacement therapies, immunomodulators (using our ISCOMATRIX® adjuvant), influenza vaccines and therapeutic proteins.
Replacement Therapies
CSL continues to enhance its immunoglobulin portfolio by expanding global registration of Privigen®, its state-of-the-art chromatographic intravenous immunoglobulin (IVIg) in the U.S. and Europe. Privigen® is a high-yield, proline-stabilised IVIg that is ready for immediate use, requiring no refrigeration or reconstitution. The product is intended for patients diagnosed with primary immunodeficiency (PI) and chronic immune thrombocytopenic purpura (ITP).
Riastap™ (human fibrinogen) was approved by the FDA in January 2009 for the treatment of patients with a congenital deficiency of this coagulation protein. It is the first and only treatment for acute bleeding episodes in patients with congential fibrinogen deficiency, an extremely rare and potentially life-threatening bleeding disorder.
On 12 October 2009, the FDA granted marketing approval for Berinert® C1-Esterase Inhibitor for the treatment of acute adominal or facial attacks associated with hereditary angioderma (HAE) a rare and serious genetic disorder. Berinert® is the first and only therapy approved for this indication in the U.S.
On 4 March 2010, the US FDA granted marketing approval for Hizentra™, Immune Globulin Subcutaneous (Human), 20% Liquid, for treating patients diagnosed with primary immunodeficiency (PI). Hizentra™ is the first 20% subcutaneous immunoglobulin (SCIg) approved in the US by the FDA. As the first SCIg treatment with a 20% concentration of immunoglobulin, Hizentra™ represents an effective, convenient choice of at-home Ig therapy that will allow people with PI to schedule treatment around their busy lives instead of scheduling their lives around treatment. Hizentra™ is an important new addition to the rapidly growing CSL Behring product portfolio, and further demonstrates our long-standing commitment to the PI and rare disease communities.
Vaccines
GARDASIL®
In 2006 Merck and Co. licensed its human papillomavirus vaccine, GARDASIL
®, in the US, Europe and Australia. This vaccine provides protection against infection by Human Papillomavirus (HPV) Types 6, 11, 16, and 18. The vaccine is based on technology licensed by CSL to Merck & Co. in 1995 which was developed as a result of collaboration in the early 1990s between CSL and Professor Ian Frazer of the University of Queensland. CSL has exclusive marketing rights for GARDASIL
® in Australia and New Zealand. Merck and Co. have recently submitted data to the US FDA for expanded use in males aged 9-26 and females aged 27-45. Merck have also commenced a phase III trial on 9-valent HPV vaccine (ie. a vaccine that provides protection against nine different HPV types). CSL's HPV patent, covering license for GARDASIL
® was also granted and will remain in effect until 2026.
Influenza Vaccines
CSL received approval from the FDA to market Afluria
®, an influenza vaccine, in the United States in September 2007. Post-licensure commitments continue in adults, the elderly and the very young. On 17 November 2009, the FDA granted approval to include active immunisation against influenza for individuals 6 months through 17 years of age.
In June 2008, following completion of successful clinical studies, CSL received approval from the Australian Therapeutic Goods Administration (TGA) for Panvax
®, CSL's "prototype" pandemic influenza vaccine based on the H5N1 strain. Panvax
® is also registered for supply after the declaration of a pandemic in Singapore.
CSL has played a key role recently in the global response to the Type A H1N1 influenza pandemic. CSL's priorities have been the rapid manufacture of vaccine and the conduct of clinical trials to support registration and supply of vaccine. In July 2009 the first of a series of clinical trials of CSL’s novel Type A (H1N1) swine influenza vaccine began. The purpose of the clinical trials was to establish an optimum vaccine dose for protection against this new strain of influenza. The data from this trial, published in the New England Journal of Medicine, showed that over 95% of participants receiving a standard single dose of vaccine achieved antibody levels that correlate with the prevention of influenza infection. Data from this and additional clinical trials has helped governments decide how best to deploy the H1N1 vaccine. CSL’s Panvax
® H1N1, a novel Type A (H1N1) influenza vaccine is now registered in Australia, the United States, Singapore and Germany. It is also registered by the World Health Organisation.
In December 2009 CSL signed an agreement with Sanofi Pasteur, the world’s leading manufacturer of vaccines, to develop a vaccine to prevent and treat periodontitis, a severe gum disease affecting approximately 30% of adults. Candidate vaccine antigens against the bacterium Porphyromonas gingivalis, which causes periodontitis, are currently being trialed in mouse models of disease. This program is being conducted within the Cooperative Research Centre for Oral Health Science.
ISCOMATRIX® Adjuvant
ISCOMATRIX
® adjuvant activities continue with our partners, progressing from pre-clinical to clinical development with several product candidates. Recently, we
also entered into a partnership with Solvay Biologicals, which has licensed CSL’s ISCOMATRIX® adjuvant.The transfer of the ISCOMATRIX
® adjuvant manufacturing process from Parkville to CSL's Kankakee, Illinois site is complete. Kankakee is now performing routine GMP manufacture of ISCOMATRIX
® adjuvant at a commercial scale.
Therapeutic Proteins
In the Therapeutic Proteins program we have progressed development of antibodies which target the IL-3 growth factor receptor for acute myeloid leukemia and the CAM3001 GM-CSFR product (partnered with AstraZeneca/MedImmune) recently entered into Phase II clinical trials.
The development of reconstituted High Density Lipoprotein (HDL) for potential use in Acute Coronary Syndrome is a priority for CSL’s R&D program. We have recently reformulated a prototype product which is now in pre-clinical testing. We are continuing studies on the biological activity and safety of CSL112 which is expected to enter the clinic in 2010.
We also have a portfolio of earlier stage projects including a project to develop therapeutic antibodies that inhibit the activity of the cytokine granulocyte colony stimulating factor (G-CSF) for the potential development of a new class of drugs that target arthritis and other inflammatory diseases.