The paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired bone marrow failure disorder, which leads to premature death and impaired red blood cells (RBCs). PNH is characterized by hemolytic anemia, thrombosis, and peripheral blood cytopenia. Mutation in the PIGA-A gene of hematopoietic stem cells causes deficiency of the two glycosylphosphatidylinositol (GPI) anchored proteins, namely CD55 and CD59 in the patient’s body, which gives rise to the premature destruction of RBCs.

Symptoms of paroxysmal nocturnal hemoglobinuria includes hemolytic anemia, hemoglobinuria, fatigue, breathlessness, abdominal pain, difficulty in controlling bleeding even from very minor wounds, appearance of small red dots on the skin that indicates bleeding under the skin, severe headache, and blood clots (thrombosis). Bone marrow transplant (stem cell transplant) is the only curative therapy for paroxysmal nocturnal hemoglobinuria available so far.

Global Paroxysmal Nocturnal Hemoglobinuria Therapeutics Market: Drivers

The global paroxysmal nocturnal hemoglobinuria therapeutics market size is expected to witness significant growth over the forecast period, owing to increasing demand for PNH therapeutics and rising number of treatment approvals from the U.S. Food and Drug Administration (FDA) for PNH, which includes iron supplements, blood transfusion, blood thinner, Eculizumab (Soliris), and bone marrow transplant. According to a study published in Blood Journal in 2012, the 5 year survival-rate of patients treated with Soliris is 95.5%.

Moreover, new R&D studies are being conducted for development of cost-effective PNH treatment, which is expected to drive the paroxysmal nocturnal hemoglobinuria therapeutics market revenue. For instance, in 2014, the research team of Perelman School of Medicine at Pennsylvania, U.S. investigated a compound named PEG-Cp40, which is long-acting and cost-effective for the treatment of paroxysmal nocturnal hemoglobinuria.

Global Paroxysmal Nocturnal Hemoglobinuria Therapeutics Market: Restraints

The paroxysmal nocturnal hemoglobinuria therapeutics market revenue is expected to hamper because of high cost for the treatment of paroxysmal nocturnal hemoglobinuria. For instance, in 2017, the American Council on Science and Health (ACSH) declared Eculizumab (Soliris) as the most expensive drug in the U.S. and cost of Solirirs ranges up to US$ 500,000 per person per year.

Moreover, the side effects shown by the drug Soliris such as headache, fatigue, nausea, vomiting, diarrhea, muscle pain, and nasal congestion can also negatively impact the paroxysmal nocturnal hemoglobinuria therapeutics market size. There are some serious side effects also, which includes low White Blood Cell (WBC) count, increased chances of bacterial infections such as meningitis, and bacterial sinusitis. According to the Centers for Disease Control and Prevention (CDC) reports of 2017, patients under the therapy of Eculizumab (Soliris) are at 1,000 to 2,000 times greater at risk of getting meningococcal diseases han the healthy individuals in the U.S.

Global Paroxysmal Nocturnal Hemoglobinuria Therapeutics Market: Regional Analysis

North America is expected to dominate in paroxysmal nocturnal hemoglobinuria therapeutics market during the forecast period, owing to new approvals from the FDA. For instance, in 2018, Ravulizumab (Ultomiris) from Alexion Pharmaceuticals and APL2 from Apellis Pharmaceuticals was approved by the FDA, for the treatment of paroxysmal nocturnal hemoglobinuria.

Asia Pacific paroxysmal nocturnal hemoglobinuria therapeutics market size is expected to witness significant growth due to availability of cost-effective treatment and medical tourism for PNH. According to an article published by National Center for Biotechnology Information (NCBI) in 2014, the cost of bone marrow transplant in India was between US$ 10,000 and US$ 40,000 in 2014 and in the U.S., it was between US$ 73,000 and US$ 140,000 in 2012.

Global Paroxysmal Nocturnal Hemoglobinuria Therapeutics Market: Competitive Landscape

Key players operating in the global paroxysmal nocturnal hemoglobinuria therapeutics market include Alexion Pharmaceuticals, Akari therapeutics, Apellis Pharmaceuticals, Amgen Inc, CinnaGen Co, Ra Pharmaceuticals Inc, Alnylam Pharmaceuticals, Achillion Pharmaceuticals Inc, Novartis, Roche, and Regeneron Pharmaceuticals.

Global Paroxysmal Nocturnal Hemoglobinuria Therapeutics Market: Taxonomy

The global paroxysmal nocturnal hemoglobinuria therapeutics market is segmented on the basis of drug type, distribution channel, and region.

By Drug Type

Eculizumab
Ravulizumab

By Distribution Channel

Hospital Pharmacy
Retail Pharmacy
Online Pharmacy

By Region

North America
Latin America
Europe
Asia Pacific
Middle East
Africa

About Author

Ghanshyam Shrivastava - With over 20 years of experience in the management consulting and research, Ghanshyam Shrivastava serves as a Principal Consultant, bringing extensive expertise in biologics and biosimilars. His primary expertise lies in areas such as market entry and expansion strategy, competitive intelligence, and strategic transformation across diversified portfolio of various drugs used for different therapeutic category and APIs. He excels at identifying key challenges faced by clients and providing robust solutions to enhance their strategic decision-making capabilities. His comprehensive understanding of the market ensures valuable contributions to research reports and business decisions.

Ghanshyam is a sought-after speaker at industry conferences and contributes to various publications on pharma industry.