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FDA clears Corifact for rare disorder

The US Food and Drug Administration has granted approval to CSL Behring's new product to prevent bleeding in people with a rare genetic disorder

The US Food and Drug Administration (FDA) has granted marketing approval to CSL Behring's new product intended to prevent bleeding in people with a rare genetic disorder.

Called Corifact (Factor XIII Concentrate [Human]), the product is indicated for the routine prophylactic treatment of congenital factor XIII (FXIII) deficiency, in which the patient's blood clots unstably, leading to recurrent bleeding, as well as problems such as poor wound healing and miscarriages. Patients are also at high risk for intracranial haemorrhage, or potentially fatal bleeding inside the skull.

Corifact is the first and only FXIII concentrate approved in the US. The treatment is already sold in 12 other countries, including the UK, under the trade name Fibrogammin.

Corifact has received the FDA's orphan drug designation, which is intended to encourage development of treatments for diseases that affect fewer than one in 200,000 Americans  and carries potential benefits including tax breaks, research grants and seven years of US marketing exclusivity. CSL Behring estimates that FXIII deficiency affects one person in two million, or about 150 people in the US.

Corifact is administered every 28 days as an intravenous infusion. The product is a fibrin-stabilising factor concentrate derived from pooled human plasma that provides both A- and B-subunits to protect against FXIII deficiency.

The treatment was approved under the FDA's accelerated approval programme, which allows for marketing clearance based on surrogate endpoints rather than demonstrated clinical benefit. A clinical study enrolling just 14 patients showed that Corifact immediately increases FXIII levels in the blood, but a direct benefit on treatment of bleeding episodes has not been established through controlled clinical trials. As a condition of approval, CSL Behring will be required to continue an ongoing study to demonstrate a clinical benefit.

"This product helps fill an important need," said Karen Midthun, director of the FDA's Center for Biologics Evaluation and Research.

21st February 2011

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