Breakthrough In Hemophilia Treatment
Hemophilia is a condition where blood does not clot, and this condition is normally inherited. The condition is caused due to defects in a gene of the X chromosome, which is a clotting factor. Generally, the diseases are widely seen in males as the X chromosome is inherited from mother to baby boy. The disease is widely treated with replacement therapy and gene therapy. The other treatment which is used is medication. However, there are ways to reduce the risk of the condition, which include regular exercise and others. The condition can be prevented by taking preventive treatment by injection of clotting factor VIII for hemophilia A, or IX for hemophilia B.
In Italy, according to the data of the National Center for Biotechnology Information (NCBI) in 2017, the number of registered people with bleeding disorders increased from about 7000 in 2000 to around 8500 in 2011 and more than 11,000 in 2015. The trend is due to an upsurge in the number of patients who are recorded, mainly in those with vWD type 1, mild hemophilia, or other factor deficiencies.
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A wide variety of hemophilia treatment is available as adjunctive measures to improve hemophilia if residual bleeding persists despite the correct application of conventional methods for hemorrhage control. Plasma-derived coagulation factor hemophilia treatment is considered to be active agents that participate in the coagulation cascade to form fibrin clots and are effective to make an intact coagulation system. Plasma is the liquid part of blood. It is pale yellow or straw-colored with proteins such as antibodies, albumin, and coagulation factors. There are several target factor drug products made from plasma proteins of human.
Since the late 1980s, hemophilia treatment has become safer and more effective. Hepatitis C virus and HIV have been eliminated from plasma-derived factors and recombinant products have been introduced for hemophilia B patients. In addition, prophylaxis has become standard for preventing bleeding instead of episodic treatment in which patients only receive clotting factors in response to a bleeding episode.
In particular, rIX-FP is a recombinant fusion protein that links FIX with recombinant albumin, a highly stable protein. Additionally, it uses the neonatal Fc receptor to prevent the protein’s degredation and induce its recycling. The Phase 3 study of rIX-FP showed a marked increase (4.3 times) in the factor half-life compared with traditional recombinant FIX product. While with twice-a-week injections of FIX concentrates the FIX lowest levels were 1% of normal levels, with rIX-FP weekly injections the FIX lowest levels were 20% and with 14-day injections they were 12% of normal levels.
In recent years, researchers have developed different technologies that prolong the half-life (durability) of the FIX proteins. In 2014, the U.S. Food and Drug Administration (FDA) approved the first of these recombinant products, which consisted of a fusion protein containing the FIX protein and a neonatal protein (neonatal Fc receptor) with increased stability. Also in 2014, Collins and colleagues published a study with the results of a Phase 3 trial assessing another recombinant FIX product, a glycoPEGylated FIX.
References:
https://www.theinsightpartners.com/reports/hemophilia-treatment-market
https://hemophilianewstoday.com/2016/08/22/new-era-hemophilia-b-treatment/