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.
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.
The report offers a comprehensive evaluation of the market. It does so via in-depth qualitative insights, historical data, and verifiable projections about market size. The projections featured in the report have been derived using proven research methodologies and assumptions. By doing so, the research report serves as a repository of analysis and information for every facet of the market, including but not limited to: Regional markets, technology, types, and applications.
The study is a source of reliable data on:
- Market segments and sub-segments
- Market trends and dynamics
- Supply and demand
- Market size
- Current trends/opportunities/challenges
- Competitive landscape
- Technological breakthroughs
- Value chain and stakeholder analysis
The regional analysis covers:
- North America (U.S. and Canada)
- Latin America (Mexico, Brazil, Peru, Chile, and others)
- Western Europe (Germany, U.K., France, Spain, Italy, Nordic countries, Belgium, Netherlands, and Luxembourg)
- Eastern Europe (Poland and Russia)
- Asia Pacific (China, India, Japan, ASEAN, Australia, and New Zealand)
- Middle East and Africa (GCC, Southern Africa, and North Africa)
The report has been compiled through extensive primary research (through interviews, surveys, and observations of seasoned analysts) and secondary research (which entails reputable paid sources, trade journals, and industry body databases). The report also features a complete qualitative and quantitative assessment by analyzing data gathered from industry analysts and market participants across key points in the industry’s value chain.
A separate analysis of prevailing trends in the parent market, macro- and micro-economic indicators, and regulations and mandates is included under the purview of the study. By doing so, the report projects the attractiveness of each major segment over the forecast period.
Highlights of the report:
- A complete backdrop analysis, which includes an assessment of the parent market
- Important changes in market dynamics
- Market segmentation up to the second or third level
- Historical, current, and projected size of the market from the standpoint of both value and volume
- Reporting and evaluation of recent industry developments
- Market shares and strategies of key players
- Emerging niche segments and regional markets
- An objective assessment of the trajectory of the market
- Recommendations to companies for strengthening their foothold in the market
Note: Although care has been taken to maintain the highest levels of accuracy in TMR’s reports, recent market/vendor-specific changes may take time to reflect in the analysis.