FDA Approves TRETTEN® for the Treatment of Congenital Factor XIII A-Subunit Deficiency

Novo Nordisk has announced the U.S. Food and Drug Administration (FDA) has approved 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.¹

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 equally². According to 2011 estimates, only 1,054 patients are diagnosed worldwide, of which an estimated 108 live in the United States³.

"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."

FDA approved TRETTEN® based on results from a clinical programme that demonstrated the safety and efficacy of TRETTEN®.¹ The phase 3 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.

About congenital FXIII A-subunit deficiency

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.

1 TRETTEN® Prescribing Information; Novo Nordisk, Inc.; Plainsboro, NJ. 2013.
2 Hsieh L, Nugent D. Factor XIII Deficiency. Haemophilia. 2008; 14: 1190-1200. Available at:
http://www.hemophilia.org/NHFWeb/Resource/StaticPages/menu0/menu5/menu330/FactorXIIIdefi
ciency.pdf. Accessed October 18, 2013.
3 World Federation of Hemophilia. Report on the Annual Global Survey 2011. WFH 2013. Available
at: http://www1.wfh.org/publications/files/pdf-1488.pdf. Accessed October 18, 2013.