Paroxysmal Nocturnal Hemoglobinuria Therapeutics Market: Competitive Intelligence and Tracking Report 2018 – 2026

Paroxysmal Nocturnal Hemoglobinuria
Therapeutics Market Predicted to Grow at a
Moderate Pace Through 2026
Paroxysmal Nocturnal Hemoglobinuria Therapeutics Market, by Drug Type
(Eculizumab and Ravulizumab), by Distribution Channel (Hospital Pharmacy,
Retail Pharmacy, and Online Pharmacy), and by Region (North America, Latin
America, Europe, Asia Pacific, Middle East, and Africa) - Size, Share, Outlook,
and Opportunity Analysis, 2018 – 2026
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. Click To Read More On Paroxysmal Nocturnal Hemoglobinuria Therapeutics Market
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.
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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: 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
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.
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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.