PNH and aHUS Market Size, Share, Growth and Industry Analysis by Type (Soliris, and, ultomiris) By Application (PNH, and, aHUS), Regional Insights, and Forecast To 2031

Updated On: 22 April 2024

PNH and aHUS Market Report Overview

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The global PNH and aHUS market size is expected to be worth USD 5151.2 million in 2021, the market is projected to touch USD 9628 million by 2031 with a CAGR of 6.4% during the forecast period.

The PNH (paroxysmal nocturnal hemoglobinuria) and aHUS (atypical hemolytic uremic syndrome) markets have witnessed significant growth in recent years. PNH is a rare hematological disorder characterized by red blood cell destruction, while aHUS are a rare kidney disease causing microvascular damage. The development of targeted therapies and advancements in diagnosis have fueled market expansion. Key players in the industry are investing in research and development to introduce innovative treatments, thus catering to the unmet medical needs of patients worldwide.

Regulatory initiatives and orphan drug designations have incentivized pharmaceutical companies to invest in research and development, leading to the development of innovative therapies for PNH and aHUS. All of the above-mentioned factors will propel the growth of the PNH and aHUS market share.

COVID-19 Impact: Increased Focus on Finding Cure for Coronavirus During Pandemic Decreased Market Growth

The pandemic of COVID-19 was the reason for imposing lockdowns and social distancing rules in order to avoid the risk of rapid spreading of the virus. The COVID-19 pandemic has had a significant impact on the management and care of patients with PNH (paroxysmal nocturnal hemoglobinuria) and aHUS (atypical hemolytic uremic syndrome). The outbreak has disrupted healthcare systems, leading to challenges in access to medical facilities and treatments for these rare diseases.

The increased focus on managing COVID-19 patients has diverted resources from other conditions, including PNH and aHUS. Additionally, individuals with PNH and aHUS may be at a higher risk of severe complications if they contract COVID-19 due to their compromised immune systems. The market experienced a downfall during the pandemic period.

LATEST TRENDS

"Development of Targeted Therapies to Accelerate Market Growth"

In recent years, there have been several notable innovations in the field of PNH (paroxysmal nocturnal hemoglobinuria) and aHUS (atypical hemolytic uremic syndrome) that have improved patient care. One significant advancement is the development of targeted therapies, such as monoclonal antibodies, which specifically target the underlying mechanisms of these diseases. These therapies have demonstrated promising results in clinical trials, providing better control of symptoms and reducing complications.

Furthermore, advancements in genetic testing and diagnosis have facilitated early detection and personalized treatment approaches. Additionally, there is ongoing research exploring novel therapeutic modalities, including gene therapy and complement inhibitors, which hold potential for further advancements in managing PNH and aHUS. All of these are considered as the latest trend in the market.

PNH and aHUS Market Segmentation

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By Type:

The market can be divided on the basis of type into the following segments:

Soliris, and, ultomiris. The soliris segment is anticipated to dominate the market during the forecast period.

By Application:

Classification based on application into the following segment:

PNH, and, aHUS. The PNH segment is predicted to dominate the market during the research period.

DRIVING FACTORS

"Incorporation of Improved Diagnostic Techniques to Fuel Market Growth"

Several driving factors contribute to the growth and development of the PNH (paroxysmal nocturnal hemoglobinuria) and aHUS (atypical hemolytic uremic syndrome) markets. Firstly, increased awareness and improved diagnostic techniques have led to better identification and understanding of these rare diseases, resulting in more accurate prevalence estimates.

Secondly, advancements in research and development have led to the introduction of targeted therapies, enhancing treatment options and improving patient outcomes. Additionally, rising healthcare expenditure, favorable reimbursement policies, and increasing investments by key market players have further propelled market growth. Also, the collaborations between pharmaceutical companies and research organizations have accelerated the development of novel therapies, driving the expansion of the PNH and aHUS market.

"Growing Awareness Among Healthcare Professionals Regarding Such Syndromes to Propel Market Growth "

In addition to the aforementioned factors, several other driving forces contribute to the growth of the PNH (paroxysmal nocturnal hemoglobinuria) and aHUS (atypical hemolytic uremic syndrome) markets. One such factor is the increasing prevalence of these diseases, which is attributed to improved diagnostic techniques and growing awareness among healthcare professionals. Moreover, patient advocacy groups and organizations play a crucial role in raising awareness and advocating for better access to treatments. Technological advancements in healthcare, including genetic testing and next-generation sequencing, have facilitated early detection and personalized treatment approaches. Furthermore, regulatory initiatives and orphan drug designations have incentivized pharmaceutical companies to invest in research and development, leading to the development of innovative therapies for PNH and aHUS. All of the above-mentioned factors are contributing towards the rapid growth and development of PNH and aHUS market growth.

RESTRAINING FACTOR

"High Cost of Targeted Therapies and Treatment Regimens to Decrease Market Growth"

While there are various driving factors, several restraining factors can impede the growth of the PNH (paroxysmal nocturnal hemoglobinuria) and aHUS (atypical hemolytic uremic syndrome) markets. One significant restraint is the high cost of targeted therapies and treatment regimens, which can limit access for patients and healthcare systems. Limited awareness among healthcare professionals and delayed diagnosis pose additional challenges in providing timely and appropriate care.

The rarity of these diseases also hinders the recruitment of patients for clinical trials and the accumulation of sufficient real-world evidence. Moreover, the regulatory complexities associated with drug development and approval processes can create barriers to market entry for potential therapies. All of these factors can bring down the market growth and development.

PNH and aHUS Market Regional Insights

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"North America to Dominate Market Share in Upcoming Years"

The leading region for the management and treatment of PNH (paroxysmal nocturnal hemoglobinuria) and aHUS (atypical hemolytic uremic syndrome) is generally determined by factors such as healthcare infrastructure, research and development activities, and market access. Currently, North America stands out as a prominent region for these rare diseases, with a well-established healthcare system, advanced diagnostic capabilities, and a high level of awareness among healthcare professionals.

several countries in this region have specialized centers and expertise in managing PNH and aHUS. Moreover, increasing focus on rare diseases in emerging markets, is likely to enhance access to diagnosis and treatment options, making them potential future leaders in addressing PNH and aHUS challenges.

Key Industry Players

"Leading Players adopt Acquisition Strategies to Stay Competitive "

Several players in the market are using acquisition strategies to build their business portfolio and strengthen their market position. In addition, partnerships and collaborations are among the common strategies adopted by companies. Key market players are making R&D investments to bring advanced technologies and solutions to the market.

List of Market Players Profiled

• Alexion Pharmaceuticals (U.S.)
• Alnylam Pharmaceuticals (U.S.)
• Omeros Corporation (U.S.)

Report Coverage

The report provides an insight into the industry from both the demand and supply sides. Further, it also gives information on the impact of COVID-19 on the market, the driving and the restraining factors along with the regional insights. Market dynamic forces during the forecast period have also been discussed for the better understanding of the market situations