Sobi announced today that their orphan drug Elocta (efmoroctocog alfa) was approved in Kuwait to treat patients with Hemophilia A.
Kuwait’s approval of the drug was based off of a Phase 3 trial which was an open-label, multi-center study involving 165 previously treated males 12 years of age and older with severe hemophilia A.
The study examined the efficacy, safety and pharmacokinetics of rFVIIIFc in previously treated males 12 years of age and older with severe hemophilia A. Each patient started on a twice-weekly dosing regimen of Elocta. Participants’ pharmacokinetic parameters were used to guide adjustments to dosing interval (every 3-5 days), and dose (25 to 65 IU/kg) to target a minimum factor VIII level of 1 to 3 IU/dL or higher as needed to prevent and control breakthrough bleeding episodes. Doses in the weekly prophylaxis arm were 65 IU/ kg/week.
The median annualized bleeding rates (ABR), including spontaneous and traumatic bleeds, were 1.6 in the individualized prophylaxis arm, 3.6 in the weekly prophylaxis arm and 33.6 in the episodic treatment arm. In the individualized prophylaxis arm, the median dosing interval was 3.5 days.
With the approval, Elocta is now the first recombinant factor VIII Fc fusion protein therapy approved for the treatment of hemophilia A in the Middle East region.
It is also approved in the European Union, Switzerland, Iceland, Liechtenstein Norway, the United States, Canada, Australia, New Zealand, Brazil, Taiwan and Japan.
Elocta fuses factor VIII to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body). By doing this, it uses a naturally occurring pathway to prolong the time the therapy remains in the body.
About Hemophilia A
Hemophilia A is a rare, chronic, genetic disorder that results in impaired clotting mechanisms due to missing or reduced levels of factor VIII.
People with hemophilia A experience recurrent and extended bleeding episodes that cause pain and irreversible joint damage. Some of these bleeding episodes can be life threatening. There are an estimated 16,000 – 20,000 people in the United States with hemophilia A (and 142,000 world wide).