Press release

Novo Nordisk receives FDA approval for REBINYN^® for routine prophylaxis to prevent bleeding in people living with Hemophilia B

PLAINSBORO, NJ, July 29, 2022 – Novo Nordisk announced today that the US Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for REBINYN^®, Coagulation Factor IX (Recombinant), GlycoPEGylated, a recombinant DNA-derived coagulation factor IX concentrate, for use in adults and children with hemophilia B (congenital factor IX deficiency) for routine prophylaxis to reduce the frequency of bleeding episodes.¹   

REBINYN^®, an extended half-life treatment used to replace clotting factor IX (FIX), was FDA approved in 2017 for on-demand treatment and control of bleeding episodes, and perioperative management of bleeding in adults and children. The expanded indication will allow for prophylactic treatment resulting in high FIX levels and reduced risk of bleeding. This is important as prophylaxis can help people with Hemophilia participate in physical and social activities with the intent to prevent bleeding.²  

“Novo Nordisk continues its long-standing commitment to drive change for the rare blood disorders community. This important milestone for REBINYN^® allows patients with Hemophilia B of all ages to maintain high factor levels until their next weekly dose.” said Melissa Leichter, Vice President & Commercial Lead, Rare Disease of Novo Nordisk.

The safety and efficacy of REBINYN^® was evaluated in five multi-center, non-controlled, open-label trials in on-demand treatment of bleeding episodes, perioperative management of major and minor surgery, and routine prophylaxis or pharmacokinetic evaluation in 115 previously treated patients (PTPs) with Hemophilia B. A total of 15,167 injections were administered over a median of 733 days, equivalent to 15,137 exposure days. This corresponds to 292 patient-years. Previously treated adults and adolescents (N=29) taking REBINYN^® 40 IU/kg once weekly for 1 year in the clinical trial program experienced an overall median annualized bleeding rate (ABR) of 1.04 along with 0 observed inhibitors or thrombotic events. Common adverse reactions (≥1%) in PTPs reported in clinical trials for REBINYN^® were itching and injection site reactions.¹

“In clinical studies, when adults and adolescent people living with Hemophilia B were given prophylactic treatment with REBINYN^®, patients experienced factor IX activity in the same range as people without hemophilia (above 40%) for nearly 80% of the week” said Allison P. Wheeler, MD, MSCI, Vanderbilt University Medical Center, Nashville, TN.  “This is supported by the World Federation of Hemophilia acknowledgement that replacement therapy with extended half-life FIX allows for more ambitious prophylaxis.”

About REBINYN^®

REBINYN^®, Coagulation Factor IX (Recombinant), GlycoPEGylated, is an extended half-life factor IX molecule for replacement therapy in patients with Hemophilia B. In five multi-center, prospective, non-controlled, open-label clinical trials, 115 PTPs received at least one dose of REBINYN^® as part of routine prophylaxis, on-demand treatment of bleeding episodes, perioperative management of major and minor surgery, or pharmacokinetic evaluation. A PTP was defined as a subject with a history of at least 150 exposure days to other Factor IX products (adolescent/adult subjects) or 50 exposure days to other Factor IX products (pediatric subjects), and no history of inhibitors. A total of 15,167 injections were administered over a median of 733 days (range: 29- 2951 days), equivalent to 15,137 exposure days and 292 patient-years.

Indications and Usage

What is Rebinyn ^® C oagulation Factor IX (Recombinant), GlycoPEGylated ?

• Rebinyn^® is an injectable medicine used to replace clotting Factor IX that is missing in patients with hemophilia B. Rebinyn^® is used to treat, prevent, or reduce the frequency (number) of bleeding episodes in people with hemophilia B. Your healthcare provider may give you Rebinyn^® when you have surgery. Rebinyn^® is not used for immune tolerance therapy.

Important Safety Information

What is the most important information I need to know about Rebinyn^®?

• Do not attempt to do an infusion yourself unless you have been taught how by your healthcare provider or hemophilia treatment center. Carefully follow your healthcare provider’s instructions regarding the dose and schedule for infusing Rebinyn^®.

Who should not use Rebinyn^®?

Do not use Rebinyn^® if you:

• are allergic to Factor IX or any of the other ingredients of Rebinyn^®.
• are allergic to hamster proteins.

What should I tell my health care provider before using Rebinyn^®?

Tell your health care provider if you:

• have or have had any medical conditions.
• take any medicines, including non-prescription medicines and dietary supplements.
• are nursing, pregnant, or plan to become pregnant.
• have been told you have inhibitors to Factor IX.

How should I use Rebinyn^®?

• Rebinyn^® is given as an infusion into the vein.
• Call your healthcare provider right away if your bleeding does not stop after taking Rebinyn^®.
• Do not stop using Rebinyn^® without consulting your healthcare provider.

What are the possible side effects of Rebinyn^®?

• Common side effects include infusion site reaction (bruising, bleeding, swelling, pain, or redness), itching, and rash.
• Your body can also make antibodies called “inhibitors” against Factor IX, including Rebinyn^®, which may stop Rebinyn^® from working properly. Your healthcare provider may need to test your blood for inhibitors from time to time.
• Call your healthcare provider right away or get emergency treatment right away if you get, for example, any of the following signs of an allergic reaction: hives, chest tightness, wheezing, difficulty breathing, and/or swelling of the face.
• You may be at an increased risk of forming blood clots in your body, especially if you have risk factors for developing blood clots. Call your healthcare provider if you have chest pain, difficulty breathing, leg tenderness or swelling.
• Animals given repeat doses of Rebinyn^® showed Polyethylene Glycol (PEG) in certain cells in the brain. The potential human implications of these animal tests are unknown.

Please see Prescribing Information for REBINYN^® at https://www.novo-pi.com/rebinyn.pdf.

About Novo Nordisk

Novo Nordisk is a leading global healthcare company that's been making innovative medicines to help people with diabetes lead longer, healthier lives for nearly 100 years. This heritage has given us experience and capabilities that also enable us to drive change to help people defeat other serious chronic diseases such as obesity and rare blood and endocrine disorders. We remain steadfast in our conviction that the formula for lasting success is to stay focused, think long-term and do business in a financially, socially, and environmentally responsible way. With U.S. headquarters in New Jersey and production and research facilities in seven states, Novo Nordisk employs nearly 6,000 people throughout the country. For more information, visit novonordisk.us, Facebook, Instagram, and Twitter.

 

Further information

Media:
Martin Havtorn Petersen	+45 3075 5246	mhpz@novonordisk.com
Natalia Salomao Abrahao (US)	+1 848-304-1027	niaa@novonordisk.com

 

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References

1. Rebinyn [package insert]. Plainsboro, NJ: Novo Nordisk Inc. 2022.
2. Srivastava A et al. Haemophilia. 2020;26(suppl 6):1-158.

 

Rebinyn^® is a registered trademark of Novo Nordisk A/S.

Novo Nordisk is a registered trademark of Novo Nordisk A/S.

© 2022 Novo Nordisk    All rights reserved.    US22REB00108  July 2022