Market Challenges And Opportunities

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: 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.