Treatment of PNH Market - Global Forecast 2026-2032

The Treatment of PNH Market size was estimated at USD 1.57 billion in 2025 and expected to reach USD 1.64 billion in 2026, at a CAGR of 4.08% to reach USD 2.08 billion by 2032.

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired hematologic disorder characterized by complement-mediated destruction of red blood cells that can lead to severe anemia, thrombosis, and impaired bone marrow function. Individuals with PNH typically present in mid-adulthood, and the disease affects approximately one to one and a half people per million globally, with median survival historically limited to a decade without targeted therapy. Despite its rarity, PNH continues to impose significant clinical burden and unmet medical need, as standard treatments have evolved from supportive care to targeted complement inhibitors to address both intravascular and extravascular hemolysis.

The therapeutic landscape for PNH has undergone dramatic transformation over the past two decades. The landmark approval of eculizumab in 2007 introduced the first terminal complement (C5) inhibitor, establishing a new standard of care that substantially reduced hemolysis and thrombotic events. Subsequent innovations have focused on enhancing treatment durability, expanding mechanism of action through proximal complement inhibition, and improving patient convenience. These advances, along with emerging oral therapies and alternative delivery modalities, have redefined treatment paradigms and created novel opportunities for improved patient outcomes and market expansion.

The introduction of terminal complement blockade represented a pivotal shift in the management of paroxysmal nocturnal hemoglobinuria. Eculizumab, approved in 2007, was the first monoclonal antibody to inhibit the C5 protein, leading to rapid and sustained reductions in intravascular hemolysis and thrombotic events. This biweekly intravenous therapy demonstrated durable efficacy in large Phase III trials, marking a major advancement over prior supportive care approaches. Building on this platform, ravulizumab emerged in 2018 as a long-acting C5 inhibitor administered every eight weeks, maintaining complete terminal complement suppression while reducing infusion frequency and enhancing patient convenience.

The next transformative wave targeted complement upstream at the level of C3. Pegcetacoplan, approved in 2021 as the first targeted C3 inhibitor, expanded therapeutic opportunity by addressing both intravascular and extravascular hemolysis. Monotherapy with this subcutaneous agent achieved statistically significant improvements in hemoglobin and transfusion avoidance compared to eculizumab, establishing proximal complement blockade as a paradigm-altering strategy in PNH care.

More recently, the advent of oral complement inhibitors has introduced unprecedented convenience and potential for more comprehensive complement control. Iptacopan, an oral factor B inhibitor, has shown near-normalization of hemoglobin and sustained transfusion independence in complement-inhibitor-naïve patients in Phase III trials, underscoring the promise of oral monotherapy in reshaping PNH treatment paradigms. In parallel, subcutaneous formulations of ravulizumab have demonstrated pharmacokinetic non-inferiority to the intravenous form, offering an alternative route of administration that may further improve quality of life and treatment adherence.

United States tariff policies enacted in 2025 have introduced significant levies on pharmaceutical imports, particularly active pharmaceutical ingredients and key intermediates, with estimated ad valorem duties ranging from 15% to 25%. Analyses by market observers have projected that these tariffs could impose a $46 billion impact on 2025 import costs, compelling manufacturers to absorb or offset increased expenditures in a sector where profit margins, especially for generic producers, are narrow. The risk of supply disruptions and elevated drug prices is acute, with generics accounting for over 90% of U.S. prescriptions yet representing only 17% of spending. Moreover, broader tariff discussions have underscored the threat of medicine shortages if manufacturers are forced to discontinue low-margin products or defer production due to cost pressures.

In response to these headwinds, forward-looking industry participants are accelerating efforts to diversify supply chains, reshore critical manufacturing capabilities, and pursue strategic partnerships to mitigate tariff exposure. Governmental stakeholders and industry associations have advocated for targeted exemptions and narrow tariff scopes to preserve essential medication availability, highlighting the need to balance national security interests with public health imperatives. At the same time, investment in domestic capacity expansion and regulatory incentives for onshore production are gaining momentum as a means to fortify resilience against external trade risks and ensure continuous patient access to life-saving therapies.

Therapeutic segmentation by treatment type reveals distinct clinical and market characteristics for each approved agent. First-generation C5 inhibition with eculizumab set the benchmark for efficacy and established the PNH treatment scaffold, while second-generation ravulizumab extended dosing intervals with an eight-week maintenance schedule. The advent of C3 blockade through pegcetacoplan introduced a complementary modality with dual intravascular and extravascular hemolysis control. These mechanistic distinctions between C5 and C3 targeting therapies underpin differentiated patient benefits and inform nuanced positioning within treatment algorithms.

Route of administration further stratifies patient care pathways and resource utilization across inpatient and outpatient channels. Intravenous infusions delivered in hospital or clinic settings have been the traditional backbone for complement therapies, whereas the approval of subcutaneous formulations and oral inhibitors enables a shift toward home-based care. Specialty clinics, and increasingly home care settings, are integrating these modalities through tailored support programs. Distribution channels, including hospital, online, and retail pharmacies, facilitate access across diverse care environments, ensuring that therapeutic innovations reach patients through optimized logistic frameworks aligned with patient preferences and healthcare system capacities.

North America remains the largest market for PNH therapies, buoyed by early adoption of novel complement inhibitors and supportive reimbursement frameworks. The U.S. Food and Drug Administration approvals for eculizumab, ravulizumab, and pegcetacoplan have enabled a robust continuum of care from infusion suites to home-administered regimens. Payers have generally embraced these high-value biologics, despite premium pricing, reflecting the critical need to mitigate PNH-related morbidity and mortality.

In Europe, orphan incentives and centralized regulatory pathways have expedited access to targeted therapies. The European Commission granted pegcetacoplan first-line indication for PNH in May 2024, following its initial approval as a second-line agent in December 2021. This extension has catalyzed broader treatment uptake across major markets such as Germany, France, and the UK, where healthcare systems leverage negotiated pricing and real-world evidence generation to optimize patient outcomes.

Asia-Pacific markets exhibit heterogeneous access dynamics. In Japan, ravulizumab received marketing authorization for PNH in June 2019, marking a milestone in reducing treatment burden through bi-monthly infusions. Elsewhere in the region, evolving regulatory frameworks and nascent reimbursement schemes are gradually accommodating novel PNH therapies, with stakeholders prioritizing local manufacturing partnerships and health technology assessments to address affordability and supply chain resilience.

Alexion Pharmaceuticals, now part of AstraZeneca, continues to leverage its Soliris franchise heritage through strategic lifecycle management and next-generation complement inhibition. Following the groundbreaking introduction of eculizumab in 2007, the company advanced ravulizumab into the market in 2018, offering an extended dosing interval of eight weeks while maintaining robust C5 blockade. Initiatives to develop subcutaneous ravulizumab formulations have further diversified patient access and administration preferences, underscoring Alexion’s commitment to customizable care models.

Apellis Pharmaceuticals, in collaboration with Sobi, has positioned pegcetacoplan as the first targeted C3 therapy for PNH, securing U.S. approval in 2021 and subsequent EU authorization for both second-line and first-line indications. The company’s integrated patient support program facilitates home infusion training and financial assistance, bolstering therapy adoption and adherence. Ongoing explorations into additional complement-driven indications reflect Apellis’s broader portfolio diversification strategy.

Novartis has emerged as a formidable competitor through the clinical development of iptacopan, an oral factor B inhibitor demonstrating unprecedented hemoglobin recovery and transfusion independence in Phase III trials. Data from the APPOINT-PNH study and subsequent APPLY-PNH extension have fueled regulatory submissions and recent FDA approval, signaling a shift toward oral monotherapy paradigms that may redefine standard-of-care considerations for treatment-naïve and anti-C5-experienced PNH populations.

Industry leaders should promptly align supply chain strategies with evolving trade policies and tariff landscapes to safeguard the availability and affordability of PNH therapies. Prioritizing localized manufacturing capacity for critical active pharmaceutical ingredients and finished drug products can mitigate tariff exposure and strengthen supply security. Engaging proactively with policymakers and industry associations to seek narrowly tailored tariff exemptions or phased implementation schedules will help maintain uninterrupted patient access. Simultaneously, expanding robust patient assistance and adherence programs will preserve market momentum and ensure continuity of care amidst cost pressures.

To drive sustained growth and clinical differentiation, biopharma stakeholders must continue advancing diversified complement inhibition approaches and delivery modalities. Strategic alliances with contract development and manufacturing organizations can accelerate the development of novel formulations, while collaborative real-world evidence initiatives will substantiate therapy value across payer and provider communities. Embracing digital health platforms for remote monitoring, telemedicine integration, and patient education will enhance patient-centricity and support long-term engagement in expanding care settings.

The analysis underpinning this executive summary synthesizes multi-source intelligence, leveraging regulatory databases, peer-reviewed literature, company disclosures, and press releases to capture the latest developments in PNH treatment. Key information was triangulated from U.S. Food and Drug Administration drug approval packages, European Medicines Agency public assessment reports, and high-visibility clinical trial publications to ensure comprehensive coverage of mechanism-specific therapies and delivery innovations. Commercial landscapes were further informed by reputable news outlets and market commentary evaluating policy shifts such as U.S. tariff impacts on pharmaceutical supply chains.

Complementing secondary research, the methodology incorporated qualitative insights from stakeholder consultations with hematology specialists, payer representatives, and supply chain managers to validate market drivers and clinical adoption trends. Iterative review cycles with internal experts and cross-functional advisory panels provided rigorous data quality checks and contextual interpretation. This blended approach guarantees a balanced perspective that integrates quantitative evidence with real-world practitioner experiences to inform strategic decision-making in the PNH treatment domain.

The evolution of PNH treatment has transitioned from broad immunosuppression to highly targeted complement inhibition, transforming patient prognosis and quality of life. Advances spanning eculizumab, ravulizumab, and pegcetacoplan have systematically addressed gaps in hemolysis control and treatment convenience, while emergent oral and subcutaneous modalities promise further gains in patient autonomy and therapy engagement. However, external pressures such as shifting trade policies and reimbursement hurdles underscore the ongoing need for agile market strategies and supply chain resilience.

Looking ahead, integrated stakeholder collaboration will be essential to optimize the full potential of next-generation therapies. By aligning clinical innovation with robust commercial frameworks and patient-centric service models, industry participants can navigate complex regulatory environments and accelerate the translation of scientific breakthroughs into sustainable, high-value care pathways. The PNH landscape is primed for further advancements, and decisive actions today will chart the course for tomorrow’s treatment standards.

For personalized guidance on leveraging these insights and to secure the full market research report, decision-makers are invited to connect directly with Ketan Rohom, Associate Director of Sales & Marketing. Engage with Ketan to access tailored strategic briefings, explore collaboration opportunities, and acquire actionable intelligence that will empower your organization to lead in the dynamic PNH treatment landscape.