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

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Dec 23, 2013, 04:10 ET

PLAINSBORO, N.J., Dec. 23, 2013 /PRNewswire/ -- 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.

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.

About TRETTEN^® (Coagulation Factor XIII A-Subunit [Recombinant])

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^®:
- Signs of allergic reaction, including shortness of breath, rash, itching (pruritus), redness of the skin (erythema), and fainting/dizziness.
- 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.
- 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.

Please visit TRETTEN-US.com for Prescribing Information.

Headquartered in Denmark, Novo Nordisk is a global healthcare company with 90 years of innovation and leadership in diabetes care. The company also has leading positions within haemophilia care, growth hormone related disorders and women's health. Novo Nordisk employs approximately 36,000 employees in 75 countries, and markets its products in more than 180 countries. For more information, visit novonordisk.com.

¹ Hsieh L, Nugent D. Factor XIII Deficiency. Haemophilia. 2008; 14: 1190-1200. Available at: http://www.hemophilia.org/NHFWeb/Resource/StaticPages/menu0/menu5/menu330/FactorXIIIdeficiency.pdf.

^²World Federation of Hemophilia. Report on the Annual Global Survey 2011. WFH 2013. Available at: http://www1.wfh.org/publications/files/pdf-1488.pdf.

SOURCE Novo Nordisk