Global Paroxysmal NocturnalHemoglobinuria Therapeutics Market: Drivers
The global ParoxysmalNocturnal Hemoglobinuria Therapeutics Market size is expected to witnesssignificant growth over the forecast period, owing to increasing demand for PNHtherapeutics and rising number of treatment approvals from the U.S. Food andDrug Administration (FDA) for PNH, which includes iron supplements, bloodtransfusion, blood thinner, Eculizumab (Soliris), and bone marrow transplant.According to a study published in Blood Journal in 2012, the 5 yearsurvival-rate of patients treated with Soliris is 95.5%.
Moreover, new R&D studies arebeing conducted for development of cost-effective PNH treatment, which isexpected to drive the paroxysmal nocturnal hemoglobinuria therapeutics marketrevenue. For instance, in 2014, the research team of Perelman School ofMedicine at Pennsylvania, U.S. investigated a compound named PEG-Cp40, which islong-acting and cost-effective for the treatment of paroxysmal nocturnalhemoglobinuria.
The paroxysmal nocturnalhemoglobinuria (PNH) is a rare, acquired bone marrow failure disorder, whichleads to premature death and impaired red blood cells (RBCs). PNH ischaracterized by hemolytic anemia, thrombosis, and peripheral blood cytopenia.Mutation in the PIGA-A gene of hematopoietic stem cells causes deficiency ofthe two glycosylphosphatidylinositol (GPI) anchored proteins, namely CD55 andCD59 in the patient’s body, which gives rise to the premature destruction ofRBCs.
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Symptoms of paroxysmal nocturnalhemoglobinuria 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 theskin, severe headache, and blood clots (thrombosis). Bone marrow transplant(stem cell transplant) is the only curative therapy for paroxysmal nocturnal hemoglobinuriaavailable so far.
Global Paroxysmal NocturnalHemoglobinuria Therapeutics Market: Restraints
The paroxysmal nocturnalhemoglobinuria therapeutics market revenue is expected to hamper because ofhigh cost for the treatment of paroxysmal nocturnal hemoglobinuria. Forinstance, in 2017, the American Council on Science and Health (ACSH) declaredEculizumab (Soliris) as the most expensive drug in the U.S. and cost ofSolirirs ranges up to US$ 500,000 per person per year.
Moreover, the side effects shownby the drug Soliris such as headache, fatigue, nausea, vomiting, diarrhea,muscle pain, and nasal congestion can also negatively impact the paroxysmalnocturnal hemoglobinuria therapeutics market size. There are some serious sideeffects also, which includes low White Blood Cell (WBC) count, increasedchances of bacterial infections such as meningitis, and bacterial sinusitis.According to the Centers for Disease Control and Prevention (CDC) reports of2017, patients under the therapy of Eculizumab (Soliris) are at 1,000 to 2,000times greater at risk of getting meningococcal diseases han the healthyindividuals in the U.S.
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Global Paroxysmal NocturnalHemoglobinuria Therapeutics Market: Regional Analysis
North America is expected todominate in paroxysmal nocturnal hemoglobinuria therapeutics market during theforecast period, owing to new approvals from the FDA. For instance, in 2018,Ravulizumab (Ultomiris) from Alexion Pharmaceuticals and APL2 from ApellisPharmaceuticals was approved by the FDA, for the treatment of paroxysmalnocturnal hemoglobinuria.
Asia Pacific paroxysmal nocturnalhemoglobinuria therapeutics market size is expected to witness significantgrowth due to availability of cost-effective treatment and medical tourism forPNH. According to an article published by National Center for BiotechnologyInformation (NCBI) in 2014, the cost of bone marrow transplant in India wasbetween US$ 10,000 and US$ 40,000 in 2014 and in the U.S., it was between US$ 73,000and US$ 140,000 in 2012.
Global Paroxysmal NocturnalHemoglobinuria Therapeutics Market: Competitive Landscape
Key players operating in the globalparoxysmal nocturnal hemoglobinuria therapeutics market include AlexionPharmaceuticals, Akari therapeutics, Apellis Pharmaceuticals, Amgen Inc,CinnaGen Co, Ra Pharmaceuticals Inc, Alnylam Pharmaceuticals, AchillionPharmaceuticals Inc, Novartis, Roche, and Regeneron Pharmaceuticals.
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