Rebinyn Approved in US as Routine Preventive Treatment for Hem B

Approvals supported by data from Phase 3 trials in PARADIGM clinical program

Teresa Carvalho, MS avatar

by Teresa Carvalho, MS |

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The U.S. Food and Drug Administration has approved Novo Nordisk’s Rebinyn (nonacog beta pegol) as a routine preventive treatment for bleeding episodes in adults and children with hemophilia B.

Rebinyn, sold under the brand name Refixia in the EU, is a replacement therapy that had originally been approved in the U.S. as an on-demand treatment to control and treat bleeds, as well as to manage those occurring around the time of surgery in both children and adults with hemophilia B.

This expanded approval now allows Rebinyn to be used as a routine prophylactic, or preventive, treatment to maintain high enough levels of factor IX (FIX) — the clotting protein missing in people with hemophilia B — and reduce the risk of bleeds. By doing so, the treatment is expected to enable people with hemophilia B to lead healthy and active lives, including participating in most physical and social activities.

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Rebinyn is an extended half-life product, meaning the delivered FIX was modified to last longer in the body and to reduce dosing frequency compared with other forms of replacement therapy.

“Novo Nordisk continues its long-standing commitment to drive change for the rare blood disorders community,” Melissa Leichter, vice president and commercial lead at Novo Nordisk Rare Disease, said in a press release. “This important milestone for Rebinyn allows patients with Hemophilia B of all ages to maintain high factor levels until their next weekly dose.”

Rebinyn/Refixia’s approvals were mainly supported by data from a series of Phase 3 trials belonging to the PARADIGM clinical trial program. These studies assessed the treatment’s safety and effectiveness for routine prophylaxis, on-demand treatment, and perioperative management in more than 100 previously treated boys and men with hemophilia B.

Data from one of these trials showed a reduced median annualized bleeding rate of 1.04 in patients 13 and older, who were given Rebinyn at a dose of 40 international units per kilogram of body weight over the course of a year.

“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, from Vanderbilt University Medical Center.

“This is supported by the World Federation of Hemophilia acknowledgement that replacement therapy with extended half-life FIX allows for more ambitious prophylaxis,” added Wheeler.