Novo Nordisk has received the US Food and Drug Administration (FDA) approval for TRETTEN (Coagulation Factor XIII A-Subunit [Recombinant]) for the routine prophylaxis of bleeding in people with congenital factor XIII (FXIII) A-subunit deficiency. TRETTEN is the only recombinant treatment for congenital FXIII A-subunit deficiency – a serious, rare bleeding disorder with limited treatment options.

TRETTEN demonstrated safety and efficacy, offering patients once-monthly dosing with a short infusion time.

Patients with congenital FXIII A-subunit deficiency have a lifelong susceptibility towards bleeding, including spontaneous intracranial haemorrhage. Caused by a lack of the protein clotting factor XIII (FXIII), congenital FXIII deficiency is estimated to occur in one in three to five million births in the United States and affects all ethnicities and both genders equally.

"Today marks an exciting milestone for people living with congenital FXIII deficiency, and we are proud to provide a recombinant therapy to people living with this very rare disease" said Mads Krogsgaard, chief science officer, Novo Nordisk." Through our expanding portfolio of recombinant products, we are committed to serving the haemophilia and rare bleeding disorders community."

US FDA approved TRETTEN based on results from a clinical programme that demonstrated the safety and efficacy of TRETTEN. The phase III trial that included 41 patients showed that when compared to an historic control group of individuals who did not receive routine FXIII infusions, preventive treatment with monthly 35 IU/kg TRETTEN injections significantly decreased the number of treatment-requiring bleeding episodes.1 The most common adverse reactions reported in the clinical trials (=1%) were headache, pain in the extremities, injection site pain and D-dimer increase.1

TRETTEN is expected to be available to patients in the US early 2014. TRETTEN is also approved in Canada as Tretten as well as in the EU, Switzerland and Australia as NovoThirteen. It has also been submitted for regulatory approval in a number of other countries.

Congenital FXIII A-subunit deficiency is caused by a lack of the protein clotting FXIII – the protein responsible for stabilizing the formation of a blood clot. In the absence of FXIII, a clot will still develop, but it will remain unstable and can break down more easily leading to delayed bleeding.

Novo Nordisk is a global healthcare company with a leadership in diabetes care. The company also has leading positions within haemophilia care, growth hormone therapy and hormone replacement therapy.