Rebinyn, which is administered intravenously, replaces the blood clotting factor IX that is missing in the disease.
The U.S. Food and Drug Administration approved it in May 2017 to control bleeding episodes and manage bleeding during an operation. The approval does not cover its use to prevent bleeding or as an immune tolerance therapy. An immune tolerance treatment consists of repeated infusions of a blood-clotting therapy over time to train the body to become used to it. The goal is to prevent the body from reacting to it by creating antibodies against it.
European regulators approved Rebinyn in June 2017.
“We’re excited to make this new treatment option available to the hemophilia community,” Pia D’ Urbano, the corporate vice president of biopharmaceuticals at Novo Nordisk, said in a press release. “The introduction of Rebinyn energizes us to build on our rich legacy of making innovative medicines available for people with rare bleeding disorders.”
The FDA’s decision to approve Rebinyn was based on the results of four completed clinical trials (NCT00956345, NCT01333111, NCT01386528 and NCT01395810) and the preliminary results of two ongoing trials (NCT01467427 and NCT02141074). Five of the six trials were Phase 3, while one was Phase 1.
In the Phase 3 trials, a single dose of Rebinyn almost doubled the clotting factor IX activity that was seen before the studies started. The average time it took for half the therapy to clear the body was 83 hours — an indication of its staying power. Doctors refer to this as the drug’s half-life.
“Even with significant progress in the treatment of hemophilia B, there is an ongoing need for medicines that help patients reach and maintain high Factor IX levels for a longer period of time,” said Dr. Guy Young, director of the Hemostasis and Thrombosis Program at Children’s Hospital Los Angeles. He was an investigator in three of the trials.
“For my patients, an extended half-life treatment that can quickly control a bleed with just one or two doses is an important part of hemophilia B care,” Young added.
Three Phase 3 trials tested Rebinyn as an on-demand therapy for bleeding episodes in adolescents and adults. One or two doses of Rebinyn were able to stop 98 percent of bleeds, researchers said.
Another trial dealt with Rebinyn’s ability to manage bleeding during surgery. It showed that a single dose before an operation led to a 100 percent bleeding-control success rate during the surgery.