Global Paroxysmal Nocturnal Hemoglobinuria Therapeutics Market - Insights
PNH (paroxysmal nocturnal hemoglobinuria) is an uncommon form of acquired bone marrow failure that causes premature death and red blood cell impairment (RBCs). Hemolytic anemia, thrombosis, and peripheral blood cytopenia are all symptoms of PNH. Deficiency of the two glycosylphosphatidylinositol (GPI) anchoring proteins, notably CD55 and CD59, in the patient's body results in premature RBC death due to a mutation in the PIGA-A gene of hematopoietic stem cells.
Symptoms of paroxysmal nocturnal hemoglobinuria includes blood clots (thrombosis), appearance of small red dots on the skin that indicates bleeding under the skin, abdominal pain, hemolytic anemia, difficulty in controlling bleeding even from very minor wounds, breathlessness, severe headache, fatigue. So far, the only cure for paroxysmal nocturnal hemoglobinuria is a bone marrow transplant (stem cell transplant).
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Drivers:
The global paroxysmal nocturnal hemoglobinuria therapeutics market is expected to grow significantly over the forecast period, owing to rising demand for PNH therapeutics and a growing number of FDA-approved treatments for PNH, which include iron supplements, blood transfusions, blood thinners, Eculizumab (Soliris), and bone marrow transplantation. According to a research published in the Blood Journal in 2012, individuals treated with Soliris have a 95.5 percent 5-year survival rate.
Furthermore, new R&D studies for the creation of a cost-effective PNH treatment are being done, which is likely to enhance the paroxysmal nocturnal hemoglobinuria therapeutics market revenue. For example, in 2014, the Perelman School of Medicine at the University of Pennsylvania explored PEG-Cp40, a long-acting and cost-effective molecule for the treatment of paroxysmal nocturia.
Restraints:
Because of the high cost of treating paroxysmal nocturnal hemoglobinuria, the market of paroxysmal nocturnal hemoglobinuria therapies is projected to suffer. For example, the American Council on Science and Health (ACSH) named Eculizumab (Soliris) the most costly medicine in the United States in 2017, and the cost of Soliris can reach US$ 500,000 per person per year.
Furthermore, the medicine Soliris' adverse effects, such as headache, fatigue, nausea, vomiting, diarrhoea, muscle soreness, and nasal congestion, may have a detrimental influence on the market for paroxysmal nocturnal hemoglobinuria therapies. Low White Blood Cell (WBC) count, higher likelihood of bacterial infections such as meningitis, and bacteria are only a few of the major side effects. According to 2017 studies from the Centers for Disease Control and Prevention (CDC), patients using Eculizumab (Soliris) are 1,000 to 2,000 times more likely than healthy people in the United States to contract meningococcal infections.
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Regional Analysis:
Because of fresh FDA approvals, North America is likely to lead the paroxysmal nocturnal hemoglobinuria treatments market throughout the forecast period. For example, in 2018, the FDA authorized Alexion Pharmaceuticals' Ravulizumab (Ultomiris) and Apellis Pharmaceuticals' APL2 for the treatment of paroxysmal nocturnal hemoglobinuria.
Due to the availability of cost-effective treatment and medical tourism for PNH, the Asia Pacific paroxysmal nocturnal hemoglobinuria therapeutics market is predicted to increase significantly. According to a 2014 study published by the National Center for Biotechnology Information (NCBI), the cost of a bone marrow transplant in India ranged between US$ 10,000 and US$ 40,000 in 2014, while it cost between US$ 73,000 and US$ 140,000 in the United States.
Table of Content
Global Paroxysmal Nocturnal Hemoglobinuria Therapeutics Market Research Report
Section 1: Global Paroxysmal Nocturnal Hemoglobinuria Therapeutics Industry Overview
Section 2: Global Economic Impact on Paroxysmal Nocturnal Hemoglobinuria Therapeutics Industry
Section 3: Global Market Competition by Industry Producers
Section 4: Global Productions, Revenue (Value), according to Regions
Section 5: Global Supplies (Production), Consumption, Export, Import, geographically
Section 6: Global Productions, Revenue (Value), Price Trend, Product Type
Section 7: Global Market Analysis, on the basis of Application
Section 8: Paroxysmal Nocturnal Hemoglobinuria Therapeutics Market Pricing Analysis
Section 9: Market Chain, Sourcing Strategy and Downstream Buyers
Section 10: Strategies and key policies by Distributors/Suppliers/Traders
Section 11: Key Marketing Strategy Analysis, by Market Vendors
Section 12: Market Effect Factors Analysis
Section 13: Global Paroxysmal Nocturnal Hemoglobinuria Therapeutics Market Forecast
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