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

December 30, 2013

PLAINSBORO, N.J. -- Novo Nordisk today announced the US 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, a serious, rare bleeding disorder with limited treatment options. TRETTEN® is the only recombinant treatment for congenital FXIII A-subunit deficiency. TRETTEN® was proven safe and effective, 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 hemorrhage. 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.1 As of 2011 estimates, only 1,054 patients are diagnosed worldwide, and approximately 115 live in the United States.2 Of those with congenital FXIII deficiency, approximately 95 percent of these patients have a deficiency of the A-subunit.1

"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 hemophilia and rare bleeding disorders community."

FDA approved TRETTEN® based on results from a clinical program that demonstrated the safety and efficacy of TRETTEN®. The phase 3 trial that included 41 patients showed that when compared with a 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. The most common adverse reactions reported in the clinical trials (> or = 1 percent) were headache, pain in the extremities, injection site pain, and D dimer increase.

"The FDA approval of TRETTEN® marks the culmination of years of research and development to help bring this much-needed treatment to market," said Eddie Williams, Corporate Vice President, BioPharmaceuticals, Novo Nordisk. "We continue to build on our rich hemophilia heritage and remain committed to serving the bleeding disorders community."

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