Hemophilia is a rare disease affecting more than 400,000 people worldwide. Hemophilia A is four times as common as hemophilia B, and more than half of patients with hemophilia A have a severe form of hemophilia. Males have a much higher chance of developing hemophilia because they only receive one copy of the X chromosome from their mother.
The mainstay of treatment for hemophilia is to replace the missing factor VIII or factor IX, produced using plasma-derived or recombinant methods. Some patients develop inhibitors to factor VIII or factor IX treatment and need a bypassing agent, such as factor VIIa, to resolve bleeds. Patients of hemophilia A or B often require lifelong treatment to prevent and control bleeding episodes, and this therefore represents a lucrative market opportunity.
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The current marketed products landscape comprises replacement factor therapies, such as recombinant therapies and new long-acting recombinant products. The current pipeline shows strong promise, as it shows a gradual shift from short-acting recombinant factor VIII or factor IX to long-acting recombinant factor VIII, and from factor replacement therapies to non-factor therapies. Nevertheless, significant unmet need remains for products that can avoid the development of inhibitors in patients with hemophilia.
Though late-stage pipeline for treatment of hemophilia seems promising, the main market restraint is likely to be low penetration of new long-acting recombinant therapies in India and China, owing to their high expected prices.
The Hemophilia A and B Asia-Pacific market will be valued at $699.1m and $112.9m respectively in 2022, growing from $444.9m and $76.3m in 2015, at compound annual growth rates of 6.7% and 5.8%.
How will non-factor therapies contribute to the growth?
What will be the impact of the new long-acting recombinant factor VIII and recombinant factor IX products in the hemophilia A and B market sales?
The pipeline contains a range of molecule types and molecular targets, including both those that are well established in hemophilia A and B, and novel targeted therapies.
Which molecular targets appear most frequently in the pipeline?
Will the pipeline address unmet needs such as less frequently administered therapies and risk of inhibitor development for hemophilia A and B patients?
What are the most promising first-in-class targets for hemophilia A and B?
Will the current first-in-class targets have a broader therapeutic potential across the Asia-Pacific markets?
Various drivers and barriers will influence the market over the forecast period.
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