Following achievements in Asia, Roche's PNH treatment drug PIASKY
(crovalimab) received its first FDA approval. This approval has
spiked the competition between Roche and current market leader
AstraZeneca. Additionally, the other pharma companies also geared
up to launch their respective lead assets.
LAS
VEGAS, Sept. 2, 2024 /PRNewswire/ -- Paroxysmal
nocturnal hemoglobinuria (PNH), a rare disorder, shows a triad
of features: intravascular hemolysis, thromboembolic events, and
cytopenia. Manifestations vary among patients, making
classification based on typical presentations challenging due to
the disease's unpredictable nature.
In 2023, the Paroxysmal Nocturnal Hemoglobinuria diagnosed
prevalent cases were ~12,000 cases in the 7MM, which might
reach ~13,000 cases by 2034. In the 7MM, the highest number
of paroxysmal nocturnal hemoglobinuria diagnosed prevalent cases
were observed in the US.
Until 2007, PNH was a devastating disease without treatment for
hemolysis and thrombosis, the leading cause of death in patients.
However, the last decade saw a revolutionary shift with the advent
of the anti-C5 agent eculizumab. It significantly reduced
hemolysis, decreased transfusion dependency, and, crucially,
lowered the thrombosis rate.
The disease-modifying therapeutic strategy for paroxysmal
nocturnal hemoglobinuria includes complement inhibition therapy,
with drugs like SOLIRIS, ULTOMIRIS, and EMPAVELI approved by
the FDA, and considered the gold standard. SOLIRIS, the pioneering
therapy for PNH, was succeeded by ULTOMIRIS, both developed by
Alexion Pharmaceuticals, offering C5 inhibitors as essential
therapeutic options.
Recent approvals of factor B and D inhibitors like FABHALTA
(iptacopan) in the US and VOYDEYA (danicopan) in
Japan aim to offer improved
treatment with fewer side effects.
Learn more about the FDA-approved paroxysmal nocturnal
hemoglobinuria drugs @ Drugs for Paroxysmal Nocturnal
Hemoglobinuria Treatment
The European Medicines Agency's Committee for Medicinal Products
for Human Use (CHMP) recommended crovalimab for treating PNH in
adults and adolescents aged 12 and above in June 2024, based on findings from the global
Phase III COMMODORE 2 clinical trial.
The FDA accepted a biologics license application for
crovalimab in September 2023
and subsequently approved it in June
2024 for treating PNH in adults and adolescents aged 13
years and older. Genentech, a subsidiary of Roche, has made the
drug available in the US.
In Japan, Chugai Pharmaceutical
submitted a new drug application for PiaSky to the Japan Ministry
of Health, Labour, and Welfare (MHLW) in June 2023. The drug was approved in March 2024 and launched in May 2024 for patients with PNH aged 12 years and
above.
In February 2024, China's National Medical Products
Administration (NMPA) approved crovalimab for treating PNH in
adults and adolescents aged 12 years and older who have not
previously received complement inhibitor treatment, following a
priority review that began in August
2022.
PiaSky is also undergoing review by other regulatory
authorities, including those in Europe and Taiwan.
To know more about paroxysmal nocturnal hemoglobinuria treatment
options, visit @ New Treatment for Paroxysmal Nocturnal
Hemoglobinuria
The paroxysmal nocturnal hemoglobinuria market is crowded with
so many companies working in the domain. Emerging PNH therapies
include Pozelimab (REGN3918) + Cemdisiran, OMS906, NM8074
(ruxoprubart), and others, reflecting a dynamic evolution in
treatment strategies.
Discover which therapies are expected to grab major paroxysmal
nocturnal hemoglobinuria market share @ Paroxysmal Nocturnal
Hemoglobinuria Market Report
Pozelimab is a novel, fully human monoclonal antibody
currently under investigation. It aims to inhibit complement factor
C5, thereby preventing the destruction of red blood cells
(hemolysis) responsible for symptoms in conditions like Paroxysmal
Nocturnal Haemoglobinuria (PNH) and other diseases influenced by
complement pathway activity. This IgG4 antibody binds tightly to
both wild-type and variant forms of human C5, effectively blocking
its function.
In its ongoing development, pozelimab is also being
studied in combination with Alnylam's cemdisiran, a siRNA C5
inhibitor. This investigational combination therapy is aimed at
treating various complement-mediated disorders, including PNH and
myasthenia gravis.
OMS-906 is currently being developed to treat
paroxysmal nocturnal hemoglobinuria (PNH), complement 3
glomerulopathy (C3G), idiopathic immune complex-mediated
glomerulonephritis (ICGN), and arthritis. It can be administered
either subcutaneously or intravenously. This drug candidate is a
monoclonal antibody designed to target mannan-binding lectin serine
protease 3 (MASP-3) to exert its therapeutic effects.
Discover more about drugs for paroxysmal nocturnal
hemoglobinuria in development @ Paroxysmal Nocturnal
Hemoglobinuria Clinical Trials
The anticipated launch of these emerging therapies for
paroxysmal nocturnal hemoglobinuria are poised to transform
the market landscape in the coming years. As these cutting-edge
therapies continue to mature and gain regulatory approval, they are
expected to reshape the paroxysmal nocturnal hemoglobinuria market
landscape, offering new standards of care and unlocking
opportunities for medical innovation and economic growth.
DelveInsight estimates that in 2023, the total paroxysmal
nocturnal hemoglobinuria market size in the 7MM was USD ~1.4
billion, which is expected to reach USD ~2.5 billion by
2034. This growth can be attributed to the advances in disease
mechanisms that drive new diagnostics and therapeutics, fueling
drug development. Additionally, the PNH market foresees positive
growth, propelled by increasing cases and upcoming therapies in the
forecast period.
DelveInsight's latest published market report titled as
Paroxysmal Nocturnal Hemoglobinuria Market Insight,
Epidemiology, and Market Forecast – 2034 will help you to
discover which market leader is going to capture the largest market
share. The report provides comprehensive insights into the
paroxysmal nocturnal hemoglobinuria country-specific treatment
guidelines, patient pool analysis, and epidemiology forecast to
help understand the key opportunities and assess the market's
underlying potential. The paroxysmal nocturnal
hemoglobinuria market report proffers epidemiological
analysis for the study period 2020–2034 in the 7MM segmented
into:
- Total Diagnosed Prevalent Cases of PNH
- Gender-specific Cases of PNH
The report provides an edge while developing business strategies
by understanding trends shaping and driving the 7MM paroxysmal
nocturnal hemoglobinuria market. Highlights include:
- 11-year Forecast
- 7MM Analysis
- Epidemiology-based Market Forecasting
- Historical and Forecasted Market Analysis upto 2034
- Emerging Drug Market Uptake
- Peak Sales Analysis
- Key Cross Competition Analysis
- Industry Expert's Opinion
- Access and Reimbursement
Download this paroxysmal nocturnal hemoglobinuria market
report to assess the epidemiology forecasts, understand the patient
journeys, know KOLs' opinions about the upcoming treatment
paradigms, and determine the factors contributing to the shift in
the paroxysmal nocturnal hemoglobinuria market. Also, stay
abreast of the mitigating factors to improve your market position
in the paroxysmal nocturnal hemoglobinuria therapeutic
space.
Related Reports
Paroxysmal Nocturnal Hemoglobinuria Epidemiology
Forecast
Paroxysmal Nocturnal Hemoglobinuria Epidemiology Forecast –
2032 report delivers an in-depth understanding of the
disease, historical and forecasted paroxysmal nocturnal
hemoglobinuria epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy,
France, and the United Kingdom), and Japan.
Paroxysmal Nocturnal Hemoglobinuria Pipeline
Paroxysmal Nocturnal Hemoglobinuria Pipeline Insight –
2024 report provides comprehensive insights about the
pipeline landscape, pipeline drug profiles, including clinical and
non-clinical stage products, and the key paroxysmal nocturnal
hemoglobinuria companies, including Biocad, AKARI
Therapeutics, Amgen, MorphoSys, Ra Pharmaceuticals, Alnylam
Pharmaceuticals, Arrowhead Pharmaceuticals, among others.
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market trends, market drivers, market barriers, and key aplastic
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Aplastic Anemia Pipeline Insight – 2024 report
provides comprehensive insights about the pipeline landscape,
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Elixirgen, among others.
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