FDA Approves Treatment Option for Congenital Factor XIII A-Subunit Deficiency

Novo Nordisk is pleased to announce that 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, one of the rarest inherited bleeding disorders. TRETTEN® is the only recombinant treatment for the disease. Patients with congenital FXIII A-subunit deficiency have a lifelong susceptibility towards bleeding, including spontaneous bleeding into the brain called intracranial hemorrhage, which, if not treated, could be life-threatening.1 TRETTEN® has been proven safe and effective and will offer patients once-monthly dosing with a short infusion time.
The 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 bleeding episodes that required treatment.
The approval of TRETTEN® is another example of Novo Nordisk’s commitment to bringing new treatment options to people living with rare bleeding disorders. At this time, we anticipate TRETTEN® will be available in early 2014.
We are providing this information to you in the event patients or caregivers contact you with questions about TRETTEN®.
Indications and Usage
What is TRETTEN® (Coagulation Factor XIII A-Subunit [Recombinant])?
· TRETTEN® is an injectable medicine used to prevent bleeding in adults and children who have congenital factor XIII (FXIII) A-subunit deficiency.
Important Safety Information
Who should not use TRETTEN®?
· You should not use TRETTEN® if you have ever had allergic (hypersensitivity) reactions, including severe, whole body reaction (anaphylaxis) to TRETTEN® or any of the ingredients.
What should I tell my healthcare provider before TRETTEN® is given?
Tell your healthcare provider about all of your medical conditions, including if you are pregnant, think you may be pregnant or planning to become pregnant, are breast feeding, or have a history of blood clots.
Tell your healthcare provider and pharmacist about all of the medicines you take, including all prescription and non-prescription medicines such as over-the-counter medicines, supplements, or herbal remedies.
What are the possible side effects of TRETTEN®?
· Call your healthcare provider or the emergency department right away if you have any of the following symptoms after using TRETTEN®:
o Signs of allergic reaction, including shortness of breath, rash, itching (pruritus), redness of the skin (erythema), and fainting/dizziness.
o Signs of a blood clot, including pain, swelling, warmth, redness, or a lump in your legs or arms, chest pain, or sudden severe headache and/or loss of consciousness or function.
o Unexpected bleeding.
· Other possible side effects may include pain in your arms or legs, headache, and pain at the injection site.
· These are not all the possible side effects of TRETTEN®. Tell your healthcare provider about any side effect that bothers you or that does not go away.