FDA Approves Hemophilia B Treatment

Article

The intravenous replacement therapy controls and prevents bleeding.

The US Food and Drug Administration (FDA) has approved a treatment for on-demand treatment and control of bleeding episodes, and the perioperative management of bleeding in hemophilia B patients.

Nonacog beta pegol (Rebinyn), a long-acting recombinant factor IX (FIX) molecule, is administered as an intravenous infusion for people with hemophilia B. It is projected to improve treatment by replacing clotting Factor IX mising in patients. It is approved for use in both children and adults with hemophilia B.

The treatment, submitted for FDA approval by Novo Nordisk, underwent Phase 3 trials with 115 patients previously treated for severe or moderately severe hemophilia B. It underwent a Blood Products Advisory Committee briefing in early April, when the FDA sought opinion as to the safety of the product and its potential risks in the pediatric and elderly populations.

In nonclinical studies, repeated treatment dosing was found to create a polyethylene glycol (PEG) accumulation in the choroid plexus. Though clinical trials did not find any safety signal that was clearly caused by PEG accumulation, it was unclear whether the clinical monitoring of neurologic function was adequate to find all relevant signs or symptoms.

Bill Breitenbach, Vice President, Biopharmaceuticals Portfolio, Novo Nordisk, thanked the committed patients that underwent clinical trials.

"We are committed to the hemophilia community and will continue on our path to bring this new extended half-life treatment to patients who need it," Breitenbach said.

The treatment is expected to be launched in the US by Novo Nordisk in the first half of 2018.

Hemophilia B, a blood clotting disorder caused by a deficiency or dysfunction by coagulation FIX, is found almost exclusively in males due to FIX being encoded in the X chromosome’s F9 gene. The disorder occurs in approximately 1 in every 25,000-30,000 male births.

The disorder is commonly defined in joint hemorrhage, which lead to permanent deformities, misalignment, loss of mobility, and uneven extremity lengths. Treatment requires regular infusions with FIX-containing preparations.

The FDA had previously approved multiple forms of hemophilia B-treatment products, including recombinant FIX, plasma-derived FIX, or plasma-derived FIX complex. There are also two long-acting FIX-fusion proteins, which allow patients to less frequently infuse in routine prophylaxis regimens.

A press release regarding the approval was made available.

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