Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market registered a valuation of USD 4.3 billion in 2024 and is projected to reach USD 18.5 billion in 2037 expanding at a profitable CAGR of 11.8% during the forecast period, i.e., 2025-2037. In 2025, the industry size of paroxysmal nocturnal hemoglobinuria (PNH) treatment is assessed at USD 4.8 billion. The market’s robust growth is attributed to the rising prevalence of rare blood disorders and advancements in therapeutic interventions, favoring companies in the healthcare sector to invest in developing, distributing, and selling advanced therapeutics for treatment. A paper published in the National Library of Medicine on inherited diseases of hemoglobin as an emerging global health burden predicted diseases such as paroxysmal nocturnal hemoglobinuria would cause a severe global health burden, especially in emerging economies.
PNH is a complement-mediated disease and requires targeted treatment. The global paroxysmal nocturnal hemoglobinuria (PNH) treatment market benefits from innovations in C5 inhibitors. For instance, in March 2020, a paper published in the National Library of Medicine stated that Crovalimab, a sequential monoclonal antibody recycling technology antibody was administered in small volumes once every 4 weeks and found complete terminal complement pathway inhibitions in patients with PNH, warranting the next phase of clinical development. An increasing rate of clinical approval for PNH treatment drugs such as Solaris (eculizumab), Ultomiris (ravulizumab), and Empaveli (pegcetacoplan) boosts the market’s growth. Market players are set to benefit from the increasing adoption of advanced diagnostic techniques assisting the early detection of PNH.
Rapid advancements in DNA sequencing assist early identification of PNH boosting the growth of the global paroxysmal nocturnal hemoglobinuria (PNH) treatment market as the demand for novel therapeutics rises. Additionally, industry stakeholders are focusing on strategic partnerships to improve distribution channels in emerging markets. A rising demand for PNH treatment has led to pharmaceutical companies pushing for regulatory approvals on novel therapies. New treatments receiving regulatory approvals boost the market’s growth as manufacturers, distributors, and end-users benefit from a positive treatment approval trend. For instance, in September 2024, Roche’s PIASKY received approval from the Food and Drug Administration (FDA) of the U.S. As global healthcare increases investment in gene therapy and precision medicine, new avenues are positioned to open in the paroxysmal nocturnal hemoglobinuria (PNH) treatment market driving the robust growth curve by the end of the forecast period.
Growth Drivers
Challenges
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Base Year |
2024 |
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Forecast Year |
2025-2037 |
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CAGR |
11.8% |
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Base Year Market Size (2024) |
USD 4.3 billion |
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Forecast Year Market Size (2037) |
USD 18.5 billion |
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Regional Scope |
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Treatment Type (Complement Inhibitors, Iron Therapy, Immunosuppressant Drugs, Bone Marrow Transplantation, Other Treatment Types)
By treatment type, the complement inhibitors segment is likely to account for around 36.1% paroxysmal nocturnal hemoglobinuria (PNH) treatment market share by 2037. The segment’s growth is attributed to the primary treatment type for PNH. Complement inhibitor therapies allow patients to improve their quality and life and reduce the need for blood transfusions, contributing to the rapid growth of the segment. Ongoing approval of add-on therapies along with complement inhibitors like ravulizumab is positioned to maintain the growth curve of the segment. For instance, in April 2024, Voydeya (Danicopan) was approved in the U.S. as an add-on therapy to ravulizumab or eculizumab for the treatment of adults diagnosed with PNH.
The iron therapy treatment type segment of the global paroxysmal nocturnal hemoglobinuria treatment sector is poised to register a robust growth curve during the forecast period owing to increasing application to treat patients experiencing chronic hemolysis leading to iron deficiency. Iron supplementation can be essential in addressing anemia caused due to the breakdown of red blood cells in patients diagnosed with PNH. For instance, as per the American Society of Hematology, pregnant women diagnosed with PNH have a greater need for iron supplementation owing to intravascular hemolysis, and in certain cases, PNH patients may require intravenous iron during pregnancy. The rising awareness of PNH treatments and early diagnosis is poised to boost the application of iron therapy to support additional complications in patients suffering from PNH.
Route of Administration (Parenteral, Oral)
By route of administration, the parenteral segment of the global paroxysmal nocturnal hemoglobinuria (PNH) treatment market is projected to increase its revenue share by the end of the forecast period. The growth of the segment is owed to applications in complement inhibitors such as ravulizumab and eculizumab. The parenteral route of administration ensures effective absorption providing immediate benefits in managing life-threatening complications. Additionally, pharmaceutical companies are seeking to improve patient convenience by releasing novel therapeutics such as crovalimab that patients can self-administer. For instance, in October 2023, the FDA approved pegcetacoplan (Empaveli) on-body injector for self -administration in PNH treatment. Trends indicate developments in subcutaneous formulations that are positioned to continue the robust growth of this segment.
Our in-depth analysis of the global paroxysmal nocturnal hemoglobinuria (PNH) treatment market includes the following segments:
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Treatment Type |
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Route of Administration |
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Age Group |
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End use |
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North America Market Forecast
North America paroxysmal nocturnal hemoglobinuria (PNH) treatment market is set to capture revenue share of over 47.2% by 2037, owing to a high adoption rate of advanced therapies and a robust regulatory framework benefiting clinical trials and approvals of new treatments. North America is the home of major pharmaceutical companies such as Alexion Pharmaceuticals, which developed Soliris, and Ultomiris benefits the adoption in North America. Additionally, supportive regulatory programs such as the RareCare Patient Assistance Program by the National Organization for Rare Disorders reimburse registration costs for workshops, conferences, and educational programs related to rare diseases, boosting awareness of PNH treatments. The favorable trends in North America are poised to continue the robust growth of the paroxysmal nocturnal hemoglobinuria (PNH) treatment market.
The U.S. holds the largest share in the paroxysmal nocturnal hemoglobinuria treatment sector. The market’s growth in the country is owed to a robust regulatory ecosystem in the country ensuring timely approval of new therapeutics. For instance, in December 2023, McKesson Corporation announced the availability of Fabhalta (Iptacopan) by Novartis after Fabhalta was approved by the FDA in the same month. Additionally, government support of precision medicine research with programs such as the Precision Medicine Initiative creates a supportive ecosystem for advanced research on PNH. As academic institutions and pharmaceutical companies collaborate to develop gene therapies, the market is poised to maintain its profitable growth.
Canada exhibits rapid growth in the paroxysmal nocturnal hemoglobinuria (PNH) treatment market of North America. The market’s growth is attributed to growing awareness of PNH treatment and better access to advanced therapies. Canada has a publicly funded healthcare system called Medicare that makes high treatment costs of PNH affordable to patients, improving access to wide demographics. Additionally, the regulatory ecosystem in the country has been proactive in approving novel therapeutics for PNH treatment which boosts the sector in Canada. For instance, in August 2024, Voydeya was approved by Health Canada as an add-on to Ultomiris and Soliris in adults diagnosed with PNH.
Europe Market Analysis
Europe is poised to register the fastest growth in the paroxysmal nocturnal hemoglobinuria (PNH) treatment market by the end of the forecast period. The market growth is due to a strong regulatory framework and push for rare disease research benefiting the market. The European Medicines Agency (EMA) has been at the forefront by approving leading therapies for PNH treatment, ensuring availability across the region, and boosting patient access to new and advanced therapeutics. For instance, in November 2020, the European Commission provided marketing authorization to Alexion for a new formulation of Ultomiris with reduced infusion time for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). France and Germany are leading the paroxysmal nocturnal hemoglobinuria (PNH) treatment market in Europe.
Germany has a large share of the paroxysmal nocturnal hemoglobinuria sector in Europe. The market in Germany benefits from its advanced healthcare infrastructure and presence of research institutions and biotech companies advancing research on rare diseases. The favorable framework for research benefits the domestic paroxysmal nocturnal hemoglobinuria (PNH) treatment market evident by a growing number of clinical trials. For instance, in April 2023, data from phase 3 APPOINT-PNH trial was presented at the 2023 annual meeting of the European Society for Blood and Marrow Transplantation (EBMT) highlighting oral iptacopan enabling an estimated 92.2% of complement inhibitor in PNH patients and achieving 2g/dl or more hemoglobin increase without the requirement of blood transfusions.
France is positioned to register rapid growth in the paroxysmal nocturnal hemoglobinuria sector in Europe. The paroxysmal nocturnal hemoglobinuria (PNH) treatment market’s growth is owed to favorable healthcare frameworks, such as the French National Plan for Rare Diseases. The next National Plan for Rare Diseases in France will focus on research & development as per the government that is set to benefit the growth of the PNH treatment sector. Additionally, the French Registry of Marrow Failure Syndrome helps research institutions such as the American Society of Hematology to research molecular profiling for next-generation sequencing that can benefit the market’s growth. In May 2024, Aspaveli (Pegcetacoplan) was approved in Europe for the treatment of adult patients diagnosed with PNH who have hemolytic anemia. As research on PNH ramps up and a strong regulatory framework in Europe benefits the domestic paroxysmal nocturnal hemoglobinuria (PNH) treatment market in France, the PNH treatment sector is projected to continue its growth.
The global paroxysmal nocturnal hemoglobinuria treatment market is projected for a robust growth curve during the forecast period. Key market players are tying up with local distributors to penetrate new markets and invest in research and development to increase the efficacy of treatment.
Here are some key players in the market:
Author Credits: Radhika Pawar
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