Global Paroxysmal Nocturnal Hemoglobinuria Drug Market To Grow At A CAGR Of 12.5% By 2030

13-Jul-2023 | Zion Market Research

The global paroxysmal nocturnal hemoglobinuria drug market size was worth around USD 3500 million in 2022 and is predicted to grow to around USD 8900 million by 2030 with a compound annual growth rate (CAGR) of roughly 12.5% between 2023 and 2030.

Global Paroxysmal Nocturnal Hemoglobinuria Drug Market Size

The paroxysmal nocturnal hemoglobinuria (PNH) drug market refers to the pharmaceutical industry's segment that focuses on the development, production, and distribution of medications for the treatment of PNH. PNH is a rare and potentially life-threatening blood disorder characterized by the abnormal breakdown of red blood cells, leading to a range of symptoms such as anemia, blood clots, and organ damage. The introduction of targeted therapies has revolutionized the management of PNH, providing improved outcomes and quality of life for patients. Eculizumab (trade name Soliris) was the first targeted therapy approved for PNH and has been a cornerstone of treatment for many years. It works by inhibiting the complement system, which is responsible for the destruction of red blood cells in PNH. Ravulizumab (trade name Ultomiris) is a more recent addition to the PNH treatment landscape, offering an extended dosing interval compared to eculizumab. The PNH drug market is driven by several factors, including the growing prevalence of PNH, increased awareness and diagnosis, advancements in drug research & development, and favorable regulatory environments. However, the market also faces challenges such as high treatment costs, limited patient population, regulatory & reimbursement barriers, and the need for alternative treatment options. Pharmaceutical companies, healthcare providers, patient advocacy groups, and regulatory bodies play crucial roles in shaping the PNH drug market. Ongoing research and development efforts aim to further enhance treatment options, develop alternative therapies, and improve patient outcomes. The paroxysmal nocturnal hemoglobinuria drug market's future outlook is influenced by factors such as emerging therapies, evolving healthcare policies, and advancements in diagnostic capabilities.

This review is based on a report by Zion Market Research, titled "Paroxysmal Nocturnal Hemoglobinuria Drug Market By Treatment (Medication, Supplements, Blood Transfusion, And Bone Marrow Transplant), By Drugs (Eculizumab, Ravulizumab, And Others), By Route Of Administration (Oral And Parenteral), By Distribution Channel (Direct, Online Pharmacy, Retailers, And Others), By End User (Hospitals, Homecare, Specialty Clinics, And Others) And By Region - Global And Regional Industry Overview, Market Intelligence, Comprehensive Analysis, Historical Data, And Forecasts 2023 – 2030"- Report at

The U.S. Food and Drug Administration (FDA) has approved an increasing number of PNH treatments, including iron supplements, blood transfusions, blood thinners, eculizumab (Soliris), and bone marrow transplants. This is expected to cause a significant increase in the global market for paroxysmal nocturnal hemoglobinuria (PNH) therapeutics over the forecast period. Additionally, there are ongoing research and development projects to create paroxysmal nocturnal hemoglobinuria (PNH) medication that is affordable and is anticipated to propel the market for therapies for PNH toward growth. The market is growing as a result of the increasing frequency of blood diseases. During the projected period, the paroxysmal nocturnal hemoglobinuria drug market expansion is anticipated to be fueled by the introduction of biologics that aim to treat the underlying problem behind a hemolytic defect or an aberrant hematopoietic stem cell. The high unmet need for drugs in emerging nations is also fueling market expansion.

Further, the paroxysmal nocturnal hemoglobinuria drug industry is expanding as a result of increased government, public, and private organization investment in the discovery of innovative pharmaceuticals. The market's ability to expand is being constrained by the high cost of medication and surgical procedures such as stem cell transplantation (SCT). During the projection period, unfavorable side effects of existing therapies might restrain market expansion. An unfavorable reimbursement environment in emerging nations is impeding the market's expansion. The dangers of bone marrow transplantation are limiting market expansion.

Global paroxysmal nocturnal hemoglobinuria medication market size

The global paroxysmal nocturnal hemoglobinuria drug industry is segmented based on treatment, drugs, route of administration, distribution channel, end-user, and region.

Based on the treatment, the global market is bifurcated into medication, supplements, blood transfusion, and bone marrow transplants. The medication segment is expected to grow at the fastest rate during the forecast period. The segment growth is attributed to the approval of various types of drugs.

Based on the drugs, the global paroxysmal nocturnal hemoglobinuria drug industry is divided into eculizumab, ravulizumab, and others.

Based on the route of administration, the global market is divided into oral and parenteral.

Based on the distribution channel, the global paroxysmal nocturnal hemoglobinuria drug industry is segmented into direct, online pharmacies, retailers, and others.

Based on the end user, the global paroxysmal nocturnal hemoglobinuria drug industry is segmented into hospitals, homecare, specialty clinics, and others.

North America is expected to dominate the global paroxysmal nocturnal hemoglobinuria drug market over the forecast period. Reimbursement policies and coverage by public and private insurance systems impact the accessibility of PNH drugs in North America. The availability of reimbursement for targeted therapies varies across different U.S. states and Canadian provinces, with insurance coverage plays a crucial role in patient access and affordability. Thereby, driving the growth of the market over the forecast period.

Recent Development:

  • In April 2023, Novartis released comprehensive findings from the Phase III APPOINT-PNH study of experimental oral monotherapy iptacopan in people with paroxysmal nocturnal hemoglobinuria (PNH) who had not previously used complement inhibitors (including anti-C5 treatments). The trial exhibited clinically significant advantages across secondary goals and fulfilled its primary endpoint1. These findings were presented at the European Society for Blood and Marrow Transplantation's (EBMT) 49th Annual Meeting.

Paroxysmal Nocturnal Hemoglobinuria Drug Market: Competitive Analysis

The global paroxysmal nocturnal hemoglobinuria drug market is dominated by players like:

  • Apellis Pharmaceuticals
  • Alexion Pharmaceuticals Inc
  • Akari Therapeutics Plc
  • CinnaGen Co
  • Ra Pharmaceuticals Inc
  • Amgen Inc
  • Achillion Pharmaceuticals Inc
  • Alnylam Pharmaceuticals Inc
  • F. Hoffmann-La Roche Ltd
  • Novartis AG
  • Regeneron Pharmaceuticals Inc
  • Samsung Bioepis
  • Amyndas Pharmaceuticals
  • Teva Pharmaceutical Industries Ltd.
  • LGM Pharma.
  • Lannett
  • NorthStar Rx LLC
  • Abbott
  • Cook 
  • Par Pharmaceutical

The global paroxysmal nocturnal hemoglobinuria drug market is segmented as follows:

By Treatment

  • Medication
  • Supplements
  • Blood Transfusion
  • Bone Marrow Transplant

By Drugs

  • Eculizumab
  • Ravulizumab
  • Others

By Route of Administration

  • Oral
  • Parenteral

By Distribution Channel

  • Direct
  • Online Pharmacy
  • Retailers
  • Others

By End User

  • Hospitals
  • Homecare
  • Specialty Clinics
  • Others

By Region

  • North America
    • The U.S.
    • Canada
  • Europe
    • France 
    • The UK
    • Spain
    • Germany
    • Italy
    • Rest of Europe
  • Asia Pacific
    • China
    • Japan
    • India
    • South Korea
    • Southeast Asia
    • Rest of Asia Pacific
  • Latin America
    • Brazil
    • Mexico
    • Rest of Latin America
  • Middle East & Africa
    • GCC
    • South Africa
    • Rest of Middle East & Africa

About Us:

Zion Market Research is an obligated company. We create futuristic, cutting-edge, informative reports ranging from industry reports, the company reports to country reports. We provide our clients not only with market statistics unveiled by avowed private publishers and public organizations but also with vogue and newest industry reports along with pre-eminent and niche company profiles. Our database of market research reports comprises a wide variety of reports from cardinal industries. Our database is been updated constantly in order to fulfill our clients with prompt and direct online access to our database. Keeping in mind the client’s needs, we have included expert insights on global industries, products, and market trends in this database. Last but not the least, we make it our duty to ensure the success of clients connected to us—after all—if you do well, a little of the light shines on us.

Contact Us:

Zion Market Research
244 Fifth Avenue, Suite N202
New York, 10001, United States
Tel: +49-322 210 92714
USA/Canada Toll-Free No.1-855-465-4651

Office Address

Contact Us

Zion Market Research
Tel: +1 (302) 444-0166
USA/Canada Toll Free No.+1 (855) 465-4651

Asia Pacific Office

3rd Floor, Mrunal Paradise, Opp Maharaja Hotel, Pimple Gurav, Pune 411061, Maharashtra, India
Phone No +91 7768 006 007, +91 7768 006 008

Contact #

US OFFICE NO +1 (302) 444-0166
US/CAN TOLL FREE +1 (855) 465-4651

We Are On Social

Industry Press Release

We Accept

We have secured system to process your transaction.

  • payment methods

Business Hours

Our support available to help you 24 hours a day, five days a week.

Monday - Friday: 9AM - 6PM

Saturday - Sunday: Closed