Paroxysmal Nocturnal Hemoglobinuria Market By Treatment (Blood Transfusion, Medication and Stem Cell Transplant) - Growth, Future Prospects & Competitive Analysis, 2019 – 2027

The paroxysmal nocturnal hemoglobinuria (PNH) is currently valued at US$ 2,341.4 Mn in 2018 and is set to reach US$ 6,203.0 Mn by 2027 projecting stunning growth at a compounded annual growth rate (CAGR) of 11.7% during the forecast period from 2019 to 2027. PNH is a rare disease wherein red blood cells are reported to burst prematurely. It has a global incidence rate of 1.3 cases per million people. The high treatment cost and mortality rate associated with stem cell transplant procedures has increased the popularity of monoclonal antibodies as the most effective treatment molecule for the treatment of PNH. Presently, 16 drug molecules are being investigated under different stages of clinical trial studies which suggests strong drug pipeline during the forecast period. 

"Excellent drug safety and efficacy reported during clinical trial studies and strong product pipeline during the forecast period increase the popularity of monoclonal antibody as first line therapy for PNH"

According to the research findings brought forward by the International Bone Marrow Transplant Registry, hematopoietic stem cell transplantation employing allogeneic donors is the only curative option for patients suffering with paroxysmal nocturnal hemoglobinuria (PNH). However, this treatment module is associated with high mortality rate of 40 to 50%. Eculizumab (Soliris) developed by Alexion Pharmaceuticals, Inc., since its approval in 2007, has revolutionized the treatment regimen for PNH. It is the only licensed drug approved by the USFDA and EMA for the treatment of hemolysis associated with paroxysmal nocturnal hemoglobinuria (PNH). It is basically an anti complement monoclonal antibody which specifically targets the C5 human complement protein and inactivates it thereby increasing the survival rate of PNH patients. Excellent drug safety and efficacy reported in clinical studies and strong product pipeline during the forecast period will increase the popularity of monoclonal antibody as the first line therapy for PNH. 

"Presence of major players and affordable reimbursement scenario together drive the market growth in North America"

North America presently represents 35% market share and is spearheading the regional segment paroxysmal nocturnal hemoglobinuria (PNH) market. As per the research citing brought forward by National Institute of Health (NIH), approximately 5,000 people suffer with the disease in the United States. Affordable reimbursement scenario and presence of major players such as Amgen, Inc., Alexion Pharmaceuticals, Roche, Regeneron Pharmaceuticals etc. together drive the market growth in North America region. Europe holds 28% market growth and trails behind North America primarily due to high prevalence rate. According to the statistics provided by National Organization for Rare Disorders (NORD), the prevalence rate of PNH is 15.9 per million people in Europe. European Medical Agency has classified PNH as an orphan disease and has provided fast track designation and orphan drug status for novel drugs developed for its treatment. Asia Pacific accounts for 14.5% market growth owing to the rising public health awareness related to PNH and developing healthcare infrastructure to provide affordable treatment pertaining to PNH.

Historical & Forecast Period

This research report presents the analysis of each segment from 2017 to 2027 considering 2018 as the base year for the research. Compounded annual growth rate (CAGR) for each respective segments are calculated for the forecast period from 2019 to 2027.

Pharmaceutcials giants competing to achieve a strong foothold in the paroxysmal nocturnal Hemoglobinuria market are Amgen, Inc., Achillion Pharmaceuticals, Alexion Pharmaceuticals, Inc., Akari Therapeutics, Apellis Pharmaceuticals, Inc., CinnaGen, F.Hoffman-La Roche Ltd., Novartis AG, Ra Pharmaceuticals and Regeneron Pharmaceutcials.

Key questions answered in this report

• Which drug molecules are under clinical trial investigation and suggest to be strong drug pipeline during the forecast period?
• What is the competitive scenario currently present among major players providing treatment options for PNH?
• What is the disease etiology associated with paroxysmal nocturnal hemoglobinuria (PNH)?
• Why is eculizumab the most popular drug for the treatment of PNH?
• What is the market performance of PNH in both developed and developing regions worldwide?