Lucky 13

New treatment option for Factor XIII deficiency

Patients with factor XIII (FXIII) deficiency, the rarest bleeding dis­order—occurring in 1 in 5 million births—have a new treatment option. In December 2013, the US Food and Drug Administration (FDA) approved the use of Novo ­Nordisk’s Tretten®, the first recombinant product, to treat people with FXIII A-subunit­ deficiency­. Approximately 95% of cases are caused by a defect in subunit A.

FXIII helps stabilize blood clots. Without it, people experience prolonged nose, mouth and muscle bleeds. Women are prone to menorrhagia (heavy menstrual periods) and miscarriages. Patients are also at increased risk for intracranial hemorrhage (bleeding in the brain) that can be life-threatening.

A clinical trial of Tretten® showed that when used prophylac­tically, the treatment prevented bleeds in 90% of the 77 subjects. Side effects included headache, as well as pain at the injection site and in the extremities.

In 2011, the FDA approved CSL Behring’s Corifact®, a plasma-derived product, to treat FXIII deficiency.

Source: Novo Nordisk