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.
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.
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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: 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.
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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.
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