A New Treatment For Hemophilia B Just Became Available

According to a story from the National Hemophilia Foundation, the pharmaceutical company Novo Nordisk announced that its product REBINYN was now available for hemophilia B patients. The treatment is intended as an on-demand drug for fast acting control of bleeding episodes, and is approved for treatment for both children and adults with hemophilia B. REBINYN first received approval for public use by the FDA in May 2017.

Hemophilia is an genetic disorder that prevents the body from forming blood clots, which is essential in order to stop bleeding from wounds. People with the disorder bleed for a longer period after being injured, bruise more easily, and are more likely to experience bleeding inside the joints or in the brain. Bleeding in the joints can cause permanent damage and brain bleeding can lead to headaches, seizures, and loss of consciousness. The condition is caused by a deficiency of clotting factors. To learn more about hemophilia, click here.

It is important to note that REBINYN is only indicated for patients with hemophilia B. Hemophilia B patients suffer from a deficiency in clotting factor IX, whereas hemophilia A patients have a deficiency of clotting factor VIII. This means that a treatment that works for one type may have no effect on the other.

REBINYN is classed as a glycoPEGylated drug. PEGylated drugs use the bonding of polyethylene glycol molecules to proteins as a way to enhance the effect of the treatment. A standard treatment for hemophilia is to supplement the missing clotting factors with periodic infusions. REBINYN works by extended the half-life of clotting factor IX as it circulates. This means that patients will be protected from bleeding incidents longer and will not have to receive infusions as often.

During clinical trials, the drug was able to stop a bleeding episode in 98 percent of cases with only one or two doses. REBINYN was also effective during surgical procedures; a single dose before an operation was found to be 100 percent effective in controlling bleeding for the duration of the surgery. The treatment represents a substantial improvement for patients with hemophilia B, as the reduction in frequency of clotting factor infusions will significantly increase their quality of life.

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