Extended indication for Rebinyn could enable patients with haemophilia B to participate in physical and social activities.
FDA officials have approved Clotting Factor IX (recombinant), GlycoPEGylated (Rebinyn; Novo Nordisk) for routine prophylaxis in adults and children to prevent bleeding in individuals with hemophilia B.1
Hemophilia B is a rare genetic disorder characterized by insufficient levels of the blood protein factor IX, which is necessary for clotting. Mild cases may experience bleeding symptoms only after surgery or injury, while bleeding symptoms can occur after minor surgery or spontaneously in some moderate and most severe cases.2
The condition occurs in about 1 in 25,000 male births and is less common in females. While many women who carry the gene have no symptoms, an estimated 10% to 25% will develop mild symptoms and may develop moderate or severe symptoms.2
Rebinyn, an extended half-life treatment used to replace clotting factor IX (FIX), was approved by the FDA in 2017 for on-demand treatment and control of bleeding episodes, as well as for perioperative treatment of bleeding in adults and children. The new, expanded indication will allow for prophylactic treatment, resulting in high FIX levels and a reduced risk of bleeding. According to a press release, this is an important step because prophylaxis can allow individuals with hemophilia to participate in physical and social activities.1
“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 and commercial leader of rare diseases at Novo Nordisk, in the press release.1
The safety and efficacy of the treatment were investigated in 5 multicenter, uncontrolled, open-label studies of on-demand treatment of bleeding episodes, perioperative treatment of major and minor surgical procedures, and routine prophylaxis or pharmacokinetic evaluation in 115 previously treated patients. patients with haemophilia B. In the studies, a total of 15,167 injections were administered over a median of 733 days, corresponding to 15,137 exposure days and corresponding to 292 patient-years.1
According to the study results, treatment-experienced adults and adolescents taking Rebinyn 40 IU/kg once weekly for 1 year in the clinical study program experienced an overall median annual bleeding of 1.04 along with zero observed inhibitors or thrombotic events. Common side effects in this group were itching and injection site reactions.1
“In clinical trials, when adults and adolescents with hemophilia B received prophylactic treatment with Rebinyn, patients experienced factor IX activity in the same range as those without hemophilia (greater than 40%) for nearly 80% of the week,” says Allison P. Wheeler, MD, MSCI, said in the press release. “This is supported by the World Federation of Hemophilia’s recognition that replacement therapy with extended half-life FIX allows for more ambitious prophylaxis.”1
- Novo Nordisk Receives FDA Approval For Rebinyn For Routine Prophylaxis To Prevent Bleeding In People With Haemophilia B. Novo Nordisk; July 29, 2022. Accessed August 1, 2022. https://www.novonordisk-us.com/content/nncorp/us/en_us/media/news-archive/news-details.html?id=127305
- Hemophilia B. National Organization for Rare Disorders. Accessed August 1, 2022. https://rarediseases.org/rare-diseases/hemophilia-b/#:~:text=Factor%20IX%20is%20a%20clotting,unfected%20individuals%2C%20they%20bleed%20longer.