“With new developments in hemophilia treatment advancing at an unprecedented rate, the industry is expected to capture $16 billion in global earnings by the end of 2028” – Global Market Insights Inc.

Hemophilia treatment market trends are gaining momentum with the increasing incidence of hemophilia A and B and the overwhelming need for their treatments (Figure). According to reliable estimates, the worldwide prevalence of hemophilia A is announced at approximately 1 case per 5000 men, with nearly a third of those affected having no family history of a blood disorder. The frequency of rare diseases also varies by reporting country, with a range of 5.4 to 14.5 cases per 100,000 men.

Hemophilia is a rare disease for which approved treatment options have remained largely unchanged for some time. Recently, however, the hemophilia treatment market is seeing an explosion of innovation in treatment options under development or already approved, including certain drugs and injections.

In 2018, the FDA approved emicizumab-kxwh which was intended to reduce or prevent the frequency of bleeding episodes in people with hemophilia A. Some other options could include desmopressin, a manufactured hormone that stimulates the release of factor 8 stored, and antifibrinolytic drugs. , which prevent clots from breaking down.

Although oral medications and injections are recognized to treat the disease, its prolonged impact on human health or its side effects are not known in detail. In this case, another welcome leap into the business space is the advancement of therapeutic options.

Below are some of the latest advances in haemophilia treatment that are expected to push different economies towards healthy living and reduce the prevalence of the rare disease:

Genetical therapy

Since hemophilia is considered a genetic disease, gene therapy for its treatment could become a “possible breakthrough” in the world of health care. The treatment offers a potential cure for hemophilia patients by establishing constant endogenous expression of factor 8 or 9 after transmission of the functional gene to exchange the hemophiliac patient’s own defective gene.

While only about 25-30% of the global hemophilia population has access to factor replacement due to its expensive alternative, complications and burden, the ability of the hemophilia gene therapy solution holds out hope for a more global admission to treatment.

That said, results from one of the approach’s recent trials restored patients’ anticoagulant factor activity levels to near-normal or normal levels and reduced their annualized bleeding rates by nearly 90%.

Anti-IPTF therapy

Anti-tissue factor pathway inhibitor, or anti-TFPI, is an advanced treatment that aims to reduce bleeding by decreasing the system that prevents blood from clotting too much. This therapy restores the hemostatic balance by blocking one of the anticoagulants and preventing it from functioning normally.

Given its success, various biopharmaceutical companies are currently researching and analyzing ways to incorporate anti-TFPI therapy into their hemophilia treatment portfolio. One such company is Pfizer.

The leading pharmaceutical company recently announced the dosing of its first participant in the Phase 3 BASIS study of marstacimab, an anti-TFPI that is being evaluated for the treatment of people with severe hemophilia A or B. The results from the completed phase 2 study show that treatment with marstacimab demonstrates reductions of more than 75% in annual bleeding rates for nearly all participants in the study population.

Factor replacement therapy

Factor replacement therapy is considered one of the effective therapies for treating hemophilia. In this case, clotting factors are injected into the veins to stop severe blood loss and bleeding problems such as joints, organs and muscles. These therapies have been widely used before surgery or an operation to prevent excessive blood loss.

According to a recent study by Global Market Insights Inc., it was revealed that factor replacement therapy exceeded USD 9,412 million in 2021, in terms of valuation, across the industry. It is further expected to hold a prominent position in the world due to ongoing R&D and advancements in the space.

For example, the US FDA announced approval of factor VII therapy for hemophilia A and B with inhibitors.

In a nutshell, rising prevalence of hemophilia and growing need for related treatments globally would help the growth plot of hemophilia treatment market to rise significantly till 2028.

Reference

Hemophilia Treatment Market Size By Disease (Hemophilia A {Severe, Moderate, Mild}, Hemophilia B {Severe, Moderate, Mild}), By Product (Recombinant Factor Concentrates {Factor VIII, Factor IX}, plasma-derived factors {factor VIII, factor IX}, extended half-life products {factor VIII, factor IX}), per patient (pediatric {0-4, 5-13, 14-18}, adult {19-44 , 45+}), by treatment (prophylaxis, on demand), by therapy (factor replacement therapy, non-factor replacement therapy), by drug class (vasopressin, coagulation factors), by route of administration (injectable , nasal spray), by end use (hospitals, clinics, hemophilia treatment centers), industry analysis report, regional outlook, application potential, COVID-19 impact analysis, price trends , competitive market share and forecast, 2022-2028. Global Markets Insights website. gminsights.com/industry-analysis/hemophilia-treatment-market. Published February 2022. Accessed April 2022.