According to Biotech365, the National Hemophilia Foundation (NHF) recently revised its treatment recommendations for patients with congenital fibrinogen deficiency, or Factor I deficiency. Now, intravenously administered fibryga, developed by Swedish protein product manufacturer Octapharma, is indicated for the treatment of patients with Factor I deficiency. To read the revised guidelines, take a look at the NHF “Products Licensed” page.
Factor I Deficiency
Also known as fibrinogen deficiency, Factor I deficiency is a rare inherited blood disorder that prevents the blood from clotting properly. There are four types of Factor I deficiency: afibrinogenemia, hypofibrinogenemia, dysfibrinogenemia, and hypodysfibrinogenemia. These conditions either have no fibrinogen or varied levels of dysfunctional fibrinogen. Symptoms vary based on the specific subtype of Factor I deficiency. Those with less fibrinogen often have more severe symptoms. Symptoms of afibrinogenemia and hypofibrinogenemia include:
- Joint and muscle bleeding
- Easy bruising
- Long, heavy menstrual bleeding
- Difficulty carrying a child to term
- Gastrointestinal hemorrhage
- Bleeding from the nose, mouth, and other soft tissues
- Excessive bleeding following umbilical cord cutting or circumcision
Patients with hypodysfibrinogenemia and dysfibrogenemia may be mild or severe. Learn more about Factor I deficiency here.
Overall, fibryga is a human fibrinogen concentrate to treat bleeding in patients with Factor I deficiency. However, fibryga is not indicated for the treatment of dysfibrinogenemia. Developed by Octapharma, fibryga is a lyophilized powder. Basically, lyophilized means “freeze-dried.” So fibryga is stored as a powder either in the refrigerator or at room temperature. To prepare for intravenous use, patients combine fibryga with sterile water. Fibryga can be stored for up to 30 months (2.5 years) following its manufacturing date.
In a Phase 3 clinical trials, researchers collected extremely positive data on the efficacy of fibryga. 25 patients enrolled. These patients experienced 89 bleeding episodes and 12 surgeries, requiring 131 transfusions and other prophylactic treatment. Fibryga assisted with blood clotting, reduced bleeding episodes, and improved treatment success.