How Kogenate works
Hemophilia A is a genetic condition that causes the blood to not be able to clot. This can result in excessive bleeding both internally and externally and can be fatal if not managed. The disease is caused by a mutation in the factor VIII, or F8 gene, which encodes for a protein that normally helps blood to clot.
It is a formulation of octocog alfa, an unmodified full-length recombinant human factor VIII (FVIII) protein. Infusions of Kogenate temporarily replace the missing FVIII protein in the blood of hemophilia patients, allowing the blood to clot as normal for a period of time.
By providing FVIII protein, Kogenate allows the patients to control and prevent bleeding episodes. It can also reduce the risk of joint damage in children with hemophilia A and allows for surgery to be carried out with less risk to the patient.
Kogenate is produced by providing cells derived from hamsters and grown in the laboratory, with a copy of the human F8 gene. The cells produce the protein and this is then harvested and purified for human use.
Kogenate in clinical trials
The U.S. Food and Drug Administration (FDA) originally approved Kogenate as an on-demand treatment of hemophilia A in 1993. In June 2000, a new formulation of Kogenate in sucrose solution, called Kogenate FS, was approved. Kogenate FS reduces the intravenous infusion time, due to a higher concentration, as well as the risk of infection.
Kogenate FS was approved following a trial assessing its safety and effectiveness in 71 patients. The results, published in the scientific Journal of Thrombosis and Haemostasis, demonstrated that Kogenate FS was effective in managing bleeds in patients with hemophilia A, and was well-tolerated with no significant adverse effects.
In May 2014, Kogenate FS was approved for routine prophylaxis treatment of hemophilia A, based on the SPINART clinical trial (NCT00623480). On-demand treatment is given after bleeding has started and can’t be stopped normally, whereas prophylactic therapy is given constantly as a preventive measure. The randomized, open-label, Phase 3 clinical trial assessed the safety and efficacy of the drug in 84 male patients (ages 15-50) randomly assigned to on-demand or prophylactic Kogenate treatment for at least one year. Results published in the Journal of Thrombosis and Haemostasis demonstrated that after a median of 1.7 years of treatment, patients given prophylactic treatment had a significantly lower median number of bleed episodes per year than those given on-demand treatment. The safety profile of prophylactic Kogenate was similar to on-demand treatment.
It is administered as an intravenous infusion or through a device surgically placed under the skin. The dosage and frequency of therapy are calculated depending on the severity of the bleeding episode, or in anticipation of the type of surgery that the patient needs.
Common side effects include injection site reactions and infections from the implanted device.
Allergic reactions to Kogenate are possible especially if the patient is allergic to hamsters and other rodents. If this occurs, the treatment should be stopped immediately and a healthcare provider should be contacted. Symptoms of an allergic reaction include rash, itching, tightness of the chest or the throat, difficulty breathing, light-headedness or dizziness, nausea, and a decrease in blood pressure.
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