Fda approves additional treatments for haemophilia and inhibitors of type A or B in adults and adolescents

The U.S. Food and Drug Administration pre-approved Sevenfact (coagulation factor viia (recombinant)-jncw) on April 1 for the treatment and control of bleeding events (meso-antibodies) in adults and adolescents 12 years of age and older with type a or b hemophilia inhibitors. Sevenfact contains the active ingredients expressed in genetically modified rabbits.
"This approval provides an alternative treatment option for controlling bleeding episodes in adults and adolescents with haemophilia who have developed inhibitors," said Dr. Peter Marks, director of the FDA's Center for Biological Assessment and Research. In addition to being an important choice for patients, sevenfact is the first product for haemophilia treatment and contains active ingredients obtained from genetically modified rabbits that are genetically engineered to produce the proteins needed for clotting. This approval is an example of our efforts to advance safe biotechnology to support innovation in patient health. Hemophilia
is a congenital hemorrhagic disease caused by a dysfunction or deficiency of coagulation factor (f) viii or ix, respectively. Hemophilia patients may bleed longer than others after injury or surgery. They can also cause spontaneous bleeding in muscles, joints and organs and can be life-threatening. Individuals with inhibitors may not respond to factor replacement therapy. An estimated 20,000 people in the United States suffer from haemophilia, according to the Centers for Disease Control and Prevention (CDC). Bleeding episodes in these patients can be controlled by on-demand treatment or by using products containing fviii or fix. However, when fviii or fix inhibitors appear in these people, treating bleeding episodes with fviii or fix products may no longer be effective. In these cases, products such as Sevenfact can bypass factor viii and factor ix reactions, thus promoting blood clot formation and controlling bleeding.Sevenfact's active ingredient is a recombinant similar to human fvii, which is expressed in the breasts of genetically modified rabbits and secreted into rabbit milk. During the purification and processing of milk, fvii is converted into an active fvii (fviia). Recombined DNA (RDNA) constructs in genetically modified rabbits used to produce Sevenfact have been approved by the FDA Veterinary Center.
“ Based on a comprehensive analysis of scientific evidence, the FDA determined that RDNA constructs inserted into rabbit genome dna are safe for both animals and rabbit handlers and can effectively cause human factor vii (hfvii) proteins to be expressed in rabbit milk. Steven M. Solomon, director of the FDA's Veterinary Medicine Center, D.V.M. M.P.H. said. The FDA also determined under the National Environmental Policy Act that approval of the application would not have a significant impact on the environment.
the safety and effectiveity of Sevenfact was determined by data from a clinical study that assessed 27 patients with type A or b haemophilia using inhibitors, including 465 mild or moderate treatments and three severe bleeding episodes. The study assessed the therapeutic effect 12 hours after the initial dose. The proportion of mild or moderate bleeding episodes successfully treated at a lower dose of 75mcg / kg and at a higher dose of 225mcg / kg (no further treatment is required for bleeding episodes, no blood products were given after 12 hours and the pain did not increase) was approximately 86%. The study also included three cases of severe bleeding, all of which were successfully treated at higher doses.
Another study assessed the safety and pharmacogenetics of three incremental doses of Sevenfact in 15 patients with or without inhibitors of severe haemophilia a or b. The results of this study were used to select two doses of 75mcg / kg and 225mcg / kg assessed in the above study.
The most common side effects of Sevenfact are headache, dizziness, discomfort at the infusion site, infusion-related reactions, hematoma at the infusion site, and fever.
Sevenfact is disabled in patients who are allergic to rabbit or rabbit protein.
In patients with haemophilia a or b and with other blood clot risk factors (thrombosis), the risk of severe arterial and venous thrombosis events may increase. Hypersensitive reactions, including allergic reactions, are possible. If symptoms of thrombosis or hypersensitive reactions occur, the patient should stop Sevenfact and seek appropriate medical intervention.
The FDA approved Sevenfact's award to French laboratories and biotech companies. （cyy123.com）