FDA Panel Backs CSL Ltd Anti-Bleeding Drug Riastap

A Food and Drug Administration panel of outside medical experts Friday backed an anti-bleeding product from a subsidiary of Australia's CSL Ltd. (CSL.AU) to treat a rare blood disorder. The product, which would be sold in the U.S. under the brand name Riastap by CSL Behring, is designed to treat congenital fibrinogen deficiency, a rare bleeding disorder resulting from deficiency of fibrinogen. It's made from human plasma and would boost levels of fibrinogen. The panel unanimously said the product was safe and that it appeared to be effective at treating the blood condition. The panel's decision amounts to a recommendation that the FDA approve the product. The FDA usually follows its panels' advice but isn't required to. The agency has set a Jan. 16 deadline to act on Riastap, although it isn't clear if that deadline will be met given the proximity of the panel meeting. The company estimates only about 300 people in the U.S. have congenital fibrinogen deficiency. Fibrinogen, also known as Factor I, is a protein needed to form a blood clot. Earlier this week, the FDA released a review of Riastap, which said the product appeared to be safe and effective. The agency said clinical studies showed Riastap was effective at increasing clot firmness, but said it will require CSL to continue studying patients to see if it results in a reduction of actual bleeding events. Riastap is being considered under the FDA's accelerated review mechanism, which allows products to be approved on less data than typically required. Companies with products granted accelerated approval are required to continue studying the products and eventually submit the data to gain full or regular approval. Riastap is currently sold in 12 countries under the name Haemocomplettan P, according to the FDA.

ProFibrix Steps up Recombinant Fibrinogen Program With PER.C6(R) License From Crucell

ProFibrix B.V., today announced that it has concluded a commercial license agreement with Crucell N.V. (Euronext, NASDAQ: CRXL; Swiss Exchange: CRX) for PER.C6(R), a unique human protein production platform. The PER.C6(R) platform allows ProFibrix to manufacture recombinant human fibrinogen at levels that support the development and commercial roll-out of new products. Fibrinogen is at the heart of all ProFibrix products and is an essential part of nature's own injury-repair mechanism. The company's lead product Fibrocaps(TM) is based on fibrinogen derived from human blood plasma and is a unique dry powder topical hemostat that stops acute and severe bleeding during surgery or after trauma injury. Initially recombinant fibrinogen will be developed for systemic applications in hemostasis and later on for the development of tissue repair products.
Jaap Koopman, Ph.D., Chief Executive Officer, said: "The PER.C6(R) license gives us access to a manufacturing platform that has already been successfully tested by ProFibrix for high expression of biologically active recombinant fibrinogen. Our Chief Technology Officer Dr. Bram Bout was one of the inventors of PER.C6(R) while at Crucell. His intimate knowledge of the platform in combination with our substantial in-house expertise on the biology of fibrinogen provides us with a strong competitive position in the hemostasis market."
Jan Ohrstrom, MD, Chief Operational Officer, said: "We believe recombinant fibrinogen has the potential to become a breakthrough product in hemostasis and tissue repair. We intend to develop a systemic hemostat product based on recombinant fibrinogen to treat or prevent bleeding in patients with low fibrinogen levels."