Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare blood disease of bone marrow stem cells, which are genetically characterized by the somatic mutation in the phosphatidylinositol glycan protein A (PIG-A) gene. PNH generally occurs in the early 30s. Around 10% patients develop PNH symptoms at 21 years of age or earlier. Around 1 to 5 individuals per million people in the U.S. are estimated to suffer from PNH. This is much lower than the incidence rate of bone marrow aplasia. PNH often goes unrecognized; delay in diagnosis may range from one year to more than 10 years.
The global PNH treatment market is anticipated to expand at a rapid pace during the forecast period. It is a niche market, with many pharmaceutical and biotech companies investing in research of bone marrow stem cells. According to current studies, the ideal treatment available is to replace all the hematopoietic stem cells with normal stem cells via stem cells transplantation. However, this treatment is not ideal in some cases as stem cell transplantation requires a stable histocompatible donor. Complete stem cells transplantation is usually considered in severe cases of PNH, for instance aplastic anemia and transformation to leukemia, as these can be life threatening complications. Factors driving the PNH treatment market include rise in number of blood & bone marrow related disorders, increase in aging population, and technological advancements in stem cells transplantation. However, increase in cost of medical equipment, specifically surgical equipment required for stem cell transformation; lack of reimbursement policies in developing regions; and occurrence of side effects in related current available treatments may hamper the PNH treatment market.
The global PNH treatment market can be segmented based on diagnosis test, type of treatment, drugs, and end-user. In terms of diagnosis test, the market can be divided into complete blood count test (CBC), lactate dehydrogenase test (LDH), bilirubin test, bone marrow examination, urine test for hemosiderin, flow cytometry, and others. Based on the type of treatment, the PNH treatment market can be segregated into treatment of PNH patients associated with hemolysis, treatment of PNH patients associated with thrombosis, treatment of PNH patients associated with non-hemolytic anemia, allogeneic stem cell transplant (SCT)/bone marrow transplant (BMT), treatment of pregnant PNH patients, treatment of pediatric PNH patients, and others. In terms of drugs, the market can be classified into eculizumab (Soliris), ALXN1210, and others. Based on end-user, the PNH treatment market can be split into hospitals, pharmaceutical & biotech companies, clinics, academic & research institutes, and others.
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Geographically, the market for PNH treatment can be divided into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa (MEA). North America dominates the global PNH treatment market due to the rise in the number of blood & bone marrow related diseases, availability of satisfactory reimbursement policies, and increase in awareness about the early diagnosis of the disease in the region. The market in Europe is also expected to expand rapidly, as key players are collaborating with research institutions and labs to develop new innovative products. The PNH treatment market in Asia Pacific is anticipated to expand at a fast pace owing to the unmet needs regarding PNH treatment of the growing population. Additionally, factors such as development of the health care network, rise in disposable income, increase in health care awareness, and availability of reimbursement facilities are boosting the PNH treatment market in Asia Pacific.
Key players operating in the PNH treatment market include Alexion Pharmaceuticals, Inc., Thermo Fisher Scientific Inc., GE Healthcare, and Johnson & Johnson.