Hemophilia is an inherited bleeding disorder which prevents blood clotting. People born with hemophilia have little or no clotting factor. Clotting factor is a protein needed for normal blood clotting. There are several types of clotting factors. These proteins work with platelets to help the blood clot. About 7 out of 10 people who have hemophilia A have the severe form of the disorder. People who don't have hemophilia have a factor VIII activity of 100 percent. People who have severe hemophilia A have a factor VIII activity of less than 1 percent. Hemophilia usually occurs in males. About 1 in 5,000 males are born with hemophilia each year. There are two main types of hemophilia such as Hemophilia A which is due to factor VIII deficiency and Hemophilia B which is due to factor IX deficiency. They are clinically almost identical and are associated with spontaneous bleeding into joints, muscles and internal or external bleeding after injury or surgery.
There are three levels of Hemophilia severity like severe, moderate and mild. Severe means 0%-1% factor level: about 80% of hemophilia sufferers are considered severe. Severe hemophilia sufferers will have excessive bleeding after injuries, surgery and can also have spontaneous bleeding episodes. Moderate means 1%-5% factor level: about 10% of hemophilia sufferers are considered moderate. Moderate sufferers may have spontaneous bleeding episodes and will probably have prolonged bleeding after injuries. Mild means 5%-50% factor level: where about 10% of hemophilia sufferers are considered mild. These people have prolonged bleeding after a serious injury, trauma or surgery. In many cases, mild hemophilia is not discovered until there is excessive bleeding after a surgery or injury. In fact, it may not even be discovered until adulthood.
Though there is no cure for haemophilia, it can be controlled with regular infusions of the deficient clotting factor, i.e. factor VIII in haemophilia A or factor IX in haemophilia B. Factor replacement can be either isolated from human blood serum, recombinant, or a combination of the two. The worldwide incidence of hemophilia is estimated at more than 400,000 people. Approximately 75% of people with hemophilia around the world still receive inadequate treatment or have no access to treatment.
Growing prevalence of the disease and the increasing use of recombinant products and prophylactic regimens are expected to drive the growth of market. However, high costs associated with the hemophilia treatment limits the adoption by end-user and inhibits the growth of this market. Several advanced and effective treatments are available in the U.S., but they may require lifelong infusion of expensive drugs that are manufactured from human plasma or through recombinant biotechnology. Biogen’s Eloctate and Alprolix are competitively priced, limiting the scope of premium pricing for pipeline long-acting products. Prophylactic treatment, however, resulted in average costs of $300,000 per year.
The hemophilia management market has been segmented by drug type, by replacement therapy and by geography. The products include Eloctate which is a recombinant DNA derived, antihemophilic factor indicated in adults and children with Hemophilia A, Alprolix which is a recombinant DNA derived, coagulation factor IX concentrate indicated in adults and children with Hemophilia B. Eloctate is expected to grab more market share as the incidences of hemophilia A are more than hemophilia B. Treatment with replacement therapy involves concentrates of clotting factor VIII (for hemophilia A) or clotting factor IX (for hemophilia B) are slowly dripped or injected into a vein. These infusions help replace the clotting factor that's missing or low. Similarly replacement therapy for hemophilia A with clotting factor VIII would grow faster than the therapy for hemophilia B.
Geographically, the two major regional markets for hemophilia treatment are Europe and North America, as the prophylaxis use of this treatment is more in Europe and it is followed by North America. According to recent data by Hemophilia Federation of America, a nearly 40% of times. Western Europe uses prophylaxis nearly 50% of the time, while the U.S. provides it 33% of the time. According to CDC statistics in 2016, one in every 5,000 newborn children is affected by hemophilia A, while one in every 30,000 is suffering from hemophilia B in the U.S. At present, Asia Pacific is a lucrative market for investment, however, high cost of treatment acts as a major restraint for the market growth. Prophylaxis treatment has been the preferred treatment option for severe hemophilia across developed countries. The rest of the world witnesses sluggish growth due to lack of awareness and high treatments costs.
The major players operating in this market include Biogen, Pfizer Inc., Baxter International Inc., Bayer Pharma AG, Novo Nordisk A/S, and Grifols International SA. and CSL Behring, Octapharma are among other significant players worldwide.
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