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Swissmedic Accepts for Review CSL Behring’s MAA for rIX-FP for Patients with Hemophilia B

BERN, Switzerland — 28 July 2015

  • Upon approval, rIX-FP will offer hemophilia B patients in Switzerland prolonged dosing intervals of up to 14 days for routine prophylaxis
  • rIX-FP regulatory submissions now made in the U.S., European Economic Area and Switzerland
  • Latest milestone underscores CSL Behring’s deep commitment to developing and delivering innovative specialty biotherapies that improve the well-being of patients with serious diseases

CSL Behring announced today that Swissmedic has accepted for review a Marketing Authorization Application (MAA) for the company’s investigational long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP) for the prophylaxis and treatment of bleeding episodes in people with Hemophilia B. Upon Swissmedic approval, rIX-FP will provide hemophilia B patients with a long-acting treatment option with dosing intervals up to 14 days.

Hemophilia B is a congenital bleeding disorder characterized by deficient or defective factor IX. It affects approximately 1 in 25,000 to 50,000 people. Nearly all patients are male.

“rIX-FP is built on CSL’s strength in protein research and development and scientific leadership in bleeding disorders, coupled with a long-standing relationship with the hemophilia community,” said Dr. Andrew Cuthbertson, Chief Scientific Officer and R&D Director, CSL Limited. “Swissmedic initiating its review of the MAA for rIX-FP moves us one step closer to bringing this innovative specialty biotherapy to patients with hemophilia B in Switzerland and further underscores our deep commitment to improving the well-being of people with serious medical conditions.”

The submission is based on the PROLONG-9FP clinical development program. PROLONG-9FP includes Phase I through Phase III open-label, multicenter studies evaluating the safety and efficacy of rIX-FP in adults and children (ages 1 to 61 years) with hemophilia B (FIX ≤ 2%) who were previously treated with other factor IX products.

Results from the Phase III studies were recently presented at the 2015 International Society on Thrombosis and Haemostasis (ISTH) Congress in Toronto. The data showed median annualized spontaneous bleeding rates (AsBR) of 0.00 in patients using rIX-FP prophylactically once every 7 days (age 1-11 years) and once up to every 14 days (age 12-61 years). The data also showed that 97 percent (age 1-11 years) and 99 percent (age 12-61 years) of bleeding events were treated with one or two infusions, with the majority of events (89 percent in patients 1-11 years, and 94 percent in patients 12-61 years) treated with only one infusion. Across the Phase III studies, none of the patients developed inhibitors to factor IX or antibodies to rIX-FP. The most common adverse reaction in clinical trials was headache.

About rIX-FP

CSL Behring engineered rIX-FP to extend the half-life of recombinant factor IX through genetic fusion with recombinant albumin. CSL Behring selected recombinant albumin as its recombinant genetic fusion partner for its coagulation factor proteins due to its long physiological half-life.

Earlier this year, CSL Behring announced that the U.S. Food and Drug Administration accepted for review the company’s Biologics License Application (BLA) for rIX-FP, and the European Medicines Agency (EMA) started the Centralized Procedure for reviewing its MAA for rIX-FP.

CSL Behring’s Operations in Switzerland

In June, CSL Behring broke ground on its state-of-the-art coagulation factor plant (CSL Behring Recombinant Facility AG) in Lengnau. CSL Behring expects the plant to be fully operational by 2019. The project is expected to create at least 300 new jobs and stimulate an investment of more than 400 million Swiss Francs in the local economy over the next five years. Once operational, CSL Behring’s recombinant factor products will be manufactured there. This includes rIX-FP as well as CSL Behring’s other recombinant factor candidates in development: rVIII-SingleChain for the treatment of hemophilia A and rVIIa-FP for the treatment of hemophilia A or B with inhibitors as well congenital factor VII deficiency.

For more than 60 years, CSL Behring has been operating a research and production plant in Bern for its immunoglobulins and albumin, employing over 1,300 people.

For more information about CSL Behring’s recombinant products in development to treat hemophilia, visit http://www.cslbehring.com/products/bleeding-disorders/novel-recombinant-hemophilia-treatments.

The information in this news release is exclusively intended to inform the press. CSL Behring explicitly refers to the advertising provisions of the Swiss Pharmaceutical Advertising Law (Art. 31 and 32 of the Federal Act on Medicinal Products and Medical Devices [TPA, SR 812.21] as well as the respective regulation regarding advertising of medicinal products [AWV, SR 812.212.5]), CSL Behring especially refers to the prohibition of advertising prescription-only medicinal products directed at the general public.

About CSL Behring

The people and science of CSL Behring save lives around the world. We develop and deliver innovative specialty biotherapies, driven by our 100-year promise to help people with life-threatening conditions live full lives. With 14,000 employees and operations in 30 countries, CSL applies world-class R&D, high-quality manufacturing and patient-centered management.

CSL Behring therapies are used around the world to treat coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease, and neurological disorders in certain markets. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic disease of the newborn.

CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a global biopharmaceutical company and a member of the CSL Group of companies. The parent company, CSL Limited (ASX:CSL), is headquartered in Melbourne, Australia. For more information, visit www.cslbehring.com.

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For Inquiries within Switzerland, Contact:

Sandra Ruckstuhl
CSL Behring AG
Office: +41 (0)31 344 11 24
Mobile: +41 (0)79 544 57 49
sandra.ruckstuhl@cslbehring.com

For All Other Inquiries, Contact:

Greg Healy
CSL Behring
Office: 610-878-4841
Mobile: 610-906-4564
greg.healy@cslbehring.com

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