発作性夜間ヘモグロビン尿症（PNH）治療の世界市場

発作性夜間ヘモグロビン尿症（PNH）治療の世界市場は2030年までに84億米ドルに到達

2023年に41億米ドルと推定される発作性夜間ヘモグロビン尿症（PNH）治療の世界市場は、2023～2030年の分析期間においてCAGR 10.7%で成長し、2030年には84億米ドルに達すると予測されます。本レポートで分析したセグメントの1つである投薬治療は、CAGR 9.0%を記録し、分析期間終了時には55億米ドルに達すると予測されます。その他の治療分野の成長率は、分析期間でCAGR 14.4％と推定されます。

米国市場は11億米ドルと推定、中国はCAGR 10.1％で成長予測

米国の発作性夜間ヘモグロビン尿症（PNH）治療市場は2023年に11億米ドルと推定されます。世界第2位の経済大国である中国は、2030年までに13億米ドルの市場規模に達すると予測され、分析期間2023-2030年のCAGRは10.1%です。その他の注目すべき地域別市場としては、日本とカナダがあり、分析期間中のCAGRはそれぞれ9.1%と8.9%と予測されています。欧州では、ドイツがCAGR 7.8%で成長すると予測されています。

世界の発作性夜間ヘモグロビン尿症（PNH）治療市場- 主要動向と促進要因まとめ

発作性夜間ヘモグロビン尿症（PNH）の効果的治療が不可欠な理由とは？

発作性夜間ヘモグロビン尿症（PNH）の効果的な治療は、このまれな血液疾患に伴う重篤で生命を脅かす合併症のために不可欠です。PNHは赤血球が早期にばらばらになり、溶血性貧血や血栓症を引き起こし、重症の場合は臓器障害や死に至ります。この病気は免疫介在性の赤血球破壊を伴うため、患者は常に疲労、腹痛、呼吸困難、血栓などの衰弱症状のリスクにさらされます。治療がなければ、これらの合併症は患者のQOLに深刻な影響を及ぼし、脳卒中や臓器不全のような生命を脅かす事態を引き起こす可能性があります。PNHの治療は、溶血の予防、血栓イベントの軽減、および患者さんの転帰を改善するための症状管理に重点を置いています。

PNHの治療には通常、免疫系を標的とする治療、特に補体阻害薬が用いられ、免疫系が赤血球を攻撃するのを防ぎます。エクリズマブ（ソリリス）やラブリズマブ（ウルトミリス）のような補体阻害剤のイントロダクションは、溶血率を低下させ、血栓症のリスクを減少させることにより、PNH管理を一変させ、患者により長い余命とより良い生活の質を提供します。これらの治療は、PNHを管理し、溶血のコントロール不能に伴う重篤な合併症を予防するために不可欠なものとなっています。PNHでは血栓症、特に静脈血栓症のリスクが高いことから、補体阻害薬はこれらの合併症を減らし、患者がより安定した生活を送れるようにするために非常に重要です。

PNHの管理には、補体阻害薬に加えて、貧血を管理するための輸血や血栓形成を予防するための薬物療法などの支持療法が重要な役割を果たします。輸血はヘモグロビン濃度を維持し、疲労感や脱力感といった貧血に関連する症状に対処するのに役立ち、抗凝固薬は血栓のリスクを軽減します。PNH治療の進歩は、支持療法の必要性を減らし、補体阻害剤の有効性を向上させることに重点を置いています。PNHは複雑で危険な疾患であるため、多角的なアプローチが必要であり、効果的な治療は患者を安定させ、合併症を予防し、最終的にはPNH患者の生活の質を高めるために不可欠です。

技術的・薬理学的進歩はPNH治療をどのように変えるのか？

技術的および薬理学的進歩は、効果がより長く持続し、安全性が改善され、患者の利便性が向上した、より効果的な治療法を提供することにより、PNH治療を変革しています。PNH治療における最も重要な技術革新のひとつは、エクリズマブのような初期の治療薬よりも投与回数を減らすことができる、ラブリズマブのような長時間作用型補体阻害薬の開発です。ラブリズマブは投与間隔を2週間ごとから8週間ごとに延長し、患者に利便性を提供し、治療へのアドヒアランスを向上させる。この投与スケジュールの延長は、頻繁な通院が困難で時間のかかる患者にとって特に有益です。頻繁な点滴の負担を軽減することで、長時間作用型補体阻害薬はより管理しやすい治療ルーチンをサポートし、より患者に優しい治療経験を提供します。

C3阻害薬や代替経路阻害薬など、代替経路を標的とする新しい補体阻害薬は、既存の治療に抵抗性を示す患者に対する有望な選択肢として浮上しています。例えば、ジルコプランとペグセタコプランは、カスケードの早い段階で補体経路を遮断するように設計されており、従来のC5阻害剤に部分的あるいは不十分な反応を示す患者に有益な、より広範な阻害効果をもたらします。これらの代替経路阻害剤は、溶血をより効果的に予防し、輸血の必要性を減らすことを目的としており、現在の治療法では完全寛解に至らない患者に希望を与えるものです。これらの新しい阻害剤が臨床試験の段階に達すれば、PNH治療の選択肢を拡大し、より包括的な補体阻害を必要とする患者に解決策を提供できる可能性があります。

遺伝子治療とCRISPRに基づく調査も、PNHの長期的な解決策となりうるものとして進歩しています。まだ研究の初期段階であるが、遺伝子治療は1回限りの治療で完治させるか、継続的な投薬の必要性を大幅に減らすことを目指しています。PNHの原因となる遺伝子変異を直接標的とすることで、遺伝子治療は患者に恒久的な解決策を提供できる可能性があります。これらの技術はまだ開発中であるが、長期にわたる有効性の可能性があることから、エキサイティングな研究分野です。遺伝子治療が進歩すれば、PNH治療に革命をもたらし、生涯薬物に頼ることのない未来への希望を患者さんにもたらすかもしれないです。

患者およびヘルスケアシステムにとってのPNH治療の利点とは？

PNH治療は、症状の管理、生命を脅かす合併症のリスクの軽減、QOLの改善など、患者にとって大きな利益をもたらし、一方、ヘルスケアシステムにとっては、入院の減少や患者の予後の改善などの利益をもたらします。患者さんにとっては、エクリズマブやラブリズマブのような補体阻害薬は、溶血を減少させ、疲労、脱力感、呼吸困難などの症状を緩和することで、PNH管理を一変させました。溶血を減少させることにより、これらの治療は患者がより健康的なヘモグロビン濃度を維持するのを助け、日常活動に参加する能力を向上させる。PNH患者にとって、効果的な症状管理は、自立を維持し、日常生活への影響を軽減するために極めて重要です。

補体阻害薬はまた、PNH患者の主要な死亡原因の一つである血栓症のリスクを軽減する上で重要な役割を果たしています。血栓形成を予防することにより、これらの治療は脳卒中、肺塞栓症、臓器障害などの重篤な合併症の可能性を減少させる。未治療の血栓症は致命的な転帰をたどる可能性があるため、このような血栓イベントの減少は患者の長期生存にとって特に有益です。補体阻害薬による効果的な治療は、このように緊急介入の必要性を減らし、患者の健康状態を安定させ、長期予後を改善します。

ヘルスケアシステムにとって、PNH治療は頻繁な入院や救急治療の必要性を減らし、合併症管理に関連する医療費全体を削減します。ravulizumabのような治療薬では、投与回数が少なくて済むため、通院や点滴の頻度が減り、医療資源が確保され、コスト効率が向上します。効果的で長時間作用する解決策を提供することで、ヘルスケアシステムは急性期の介入よりも予防医療に集中することができ、患者の転帰を改善することができます。さらに、新しい治療がPNH管理の有効性を向上させるにつれ、患者は輸血や抗凝固剤などの支持療法に頼ることが少なくなり、医療費と資源配分がさらに削減されます。

さらに、臨床試験において経口補体阻害薬が開発されれば、特に輸液センターへのアクセスが制限されている地域では、患者にとって治療の利便性とアクセスがさらに向上する可能性があります。経口療法により、患者は自宅で病状を管理することができるようになり、ヘルスケア施設に頻繁に出向く必要がなくなります。このようなアクセスの良さは、農村部や十分な治療を受けていない人々にとって、移動に伴う物流や経済的負担なしに必要な治療を受けられるという大きな利点となります。ヘルスケアシステムにとって、経口療法は輸液センターや病院のリソースの需要を減らし、効率的なケア提供をさらに支援し、すべてのPNH患者にとってのアクセシビリティを向上させる。

何がPNH治療市場の成長を促進しているのか？

PNH治療市場の成長の原動力となっているのは、希少疾患に対する認識の高まり、標的治療の進歩、新しい治療法への患者アクセスの拡大、遺伝子治療研究の台頭です。ヘルスケアプロバイダーや患者の間でPNHに対する認識や理解が深まるにつれ、診断率が向上し、より早く正確な治療が開始されるようになりました。患者がより早く診断されれば、合併症を減らし、長期的な転帰を改善する救命治療を受け始めることができます。また、患者支援団体による啓発活動がPNH研究への投資を促し、先進的な治療法の開発や治療選択肢の拡大につながっています。PNHに対する認識と診断が向上するにつれ、効果的なPNH治療に対する需要は増加の一途をたどっています。

標的治療、特に補体阻害剤の進歩は、溶血を効果的にコントロールし、血栓イベントのリスクを低減するため、大きな促進要因となっています。エクリズマブやラブリズマブのようなC5阻害薬の成功は、C3阻害薬やその他の補体経路阻害薬を含む次世代治療薬の開発に道を開いた。これらの新治療薬は治療の選択肢を広げ、既存の治療で効果が不十分な患者のアンメットニーズに応えるものです。市場の成長は、希少疾患研究への関心の高まりが製薬企業のPNHに対する革新的治療への投資を促し、さらに後押ししています。各社が新たな治療法を追求し、補体阻害剤の種類を増やしていることから、PNH治療市場は成長すると考えられます。

PNH治療への患者アクセスの拡大も、規制当局の承認や保険適用の拡大によって促進され、市場成長の原動力となっています。FDAやEMAなどの規制機関は、多くのPNH治療薬に希少疾病用医薬品（オーファンドラッグ）のステータスを付与しており、承認プロセスを合理化し、さまざまな地域での入手可能性を高めています。補体阻害剤のような高コストの治療に対する保険適用も改善され、以前は治療費を支払うことができなかった患者が、これらの治療にアクセスしやすくなっています。さらに、投与回数が少なくて済む長時間作用型治療薬の導入により、特に遠隔地や十分な治療を受けていない地域の患者にとって、治療がより便利で利用しやすくなっています。PNH治療へのアクセスが世界的に拡大するにつれて、市場は成長し、より多くの患者に行き渡り、さまざまな地域で転帰が改善されることが期待されます。

遺伝子治療研究の出現は、長期的にPNH治療市場を変革する可能性を秘めています。PNHの原因となる根本的な遺伝子変異に対処することで、遺伝子治療は、生涯にわたる投薬の必要性をなくし、1回で完治する治療法を提供できる可能性を秘めています。遺伝子治療研究はまだ初期段階にあるが、PNHの症状や合併症を長期にわたって緩和する可能性があるため、大きな関心を集めています。企業がPNH治療への応用を模索しているため、遺伝子治療やCRISPRベースのソリューションへの投資が増加しています。これらの治療が成功すれば、PNH市場に革命をもたらし、患者に恒久的な解決策を提供し、費用のかかる継続的治療への依存を減らすことができます。認知度の向上、標的治療の進歩、患者アクセスの向上、遺伝子治療研究といったこれらの要因が相まって、PNH治療市場は力強い成長を遂げ、希少疾患管理と革新的ヘルスケアにおける重要な分野としての地位を確立しつつあります。

セグメント

治療（薬物療法、その他の治療）

調査対象企業の例（全42件）

• Akari Therapeutics Plc
• Alexion Pharmaceuticals, Inc.
• Apellis Pharmaceuticals
• AstraZeneca Plc
• CANbridge Pharmaceuticals
• Chugai Pharmaceutical Co., Ltd.
• F. Hoffmann-La Roche AG
• Genentech, Inc.
• Kira Pharmaceuticals
• Novartis AG

目次

第1章 調査手法

第2章 エグゼクティブサマリー

• 市場概要
• 主要企業
• 市場動向と促進要因
• 世界市場の見通し

第3章 市場分析

• 米国
• カナダ
• 日本
• 中国
• 欧州
• フランス
• ドイツ
• イタリア
• 英国
• その他欧州
• アジア太平洋
• その他の地域

第4章 競合

Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market to Reach US$8.4 Billion by 2030

The global market for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment estimated at US$4.1 Billion in the year 2023, is expected to reach US$8.4 Billion by 2030, growing at a CAGR of 10.7% over the analysis period 2023-2030. Medication Treatment, one of the segments analyzed in the report, is expected to record a 9.0% CAGR and reach US$5.5 Billion by the end of the analysis period. Growth in the Other Treatments segment is estimated at 14.4% CAGR over the analysis period.

The U.S. Market is Estimated at US$1.1 Billion While China is Forecast to Grow at 10.1% CAGR

The Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment market in the U.S. is estimated at US$1.1 Billion in the year 2023. China, the world's second largest economy, is forecast to reach a projected market size of US$1.3 Billion by the year 2030 trailing a CAGR of 10.1% over the analysis period 2023-2030. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at a CAGR of 9.1% and 8.9% respectively over the analysis period. Within Europe, Germany is forecast to grow at approximately 7.8% CAGR.

Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market - Key Trends & Drivers Summarized

Why Is Effective Treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH) Essential?

Effective treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH) is essential due to the severe and life-threatening complications associated with this rare blood disorder. PNH causes red blood cells to break apart prematurely, leading to hemolytic anemia, thrombosis, and, in severe cases, organ damage and death. Since the disease involves immune-mediated destruction of red blood cells, it places patients at constant risk for debilitating symptoms such as fatigue, abdominal pain, difficulty breathing, and blood clots. Without treatment, these complications can severely impact patients' quality of life and may result in life-threatening events like strokes and organ failure. Treatments for PNH focus on preventing hemolysis, reducing thrombotic events, and managing symptoms to improve patient outcomes.

PNH treatment typically involves therapies that target the immune system, specifically complement inhibitors, which prevent the immune system from attacking red blood cells. The introduction of complement inhibitors like eculizumab (Soliris) and ravulizumab (Ultomiris) has transformed PNH management by reducing the rate of hemolysis and decreasing the risk of thrombosis, providing patients with longer life expectancy and better quality of life. These treatments have become essential for managing the disease and preventing the serious complications associated with uncontrolled hemolysis. Given the high risk of thrombotic events in PNH, especially in veins, complement inhibitors are critical in reducing these complications and allowing patients to lead more stable lives.

In addition to complement inhibitors, supportive care plays a significant role in managing PNH, including blood transfusions to manage anemia and medications to prevent clot formation. Blood transfusions can help maintain hemoglobin levels, addressing anemia-related symptoms like fatigue and weakness, while anticoagulants can reduce the risk of blood clots. Advances in PNH treatment focus on reducing the need for supportive care and improving the efficacy of complement inhibitors. As the disease's complexity and risks require a multifaceted approach, effective treatments are vital for stabilizing patients, preventing complications, and ultimately enhancing the quality of life for those affected by PNH.

How Are Technological and Pharmacological Advancements Transforming PNH Treatment?

Technological and pharmacological advancements are transforming PNH treatment by offering more effective therapies with longer-lasting effects, improved safety, and enhanced patient convenience. One of the most significant innovations in PNH treatment is the development of long-acting complement inhibitors, such as ravulizumab, which allow for less frequent dosing than earlier treatments like eculizumab. Ravulizumab extends the dosing interval from every two weeks to every eight weeks, providing patients with greater convenience and improving adherence to treatment. This extended dosing schedule is especially beneficial for patients who find frequent hospital visits challenging and time-consuming. By reducing the burden of frequent infusions, long-acting complement inhibitors support more manageable treatment routines and provide a more patient-friendly experience.

New complement inhibitors targeting alternative pathways, such as C3 inhibitors and alternative pathway inhibitors, are emerging as promising options for patients who are resistant to existing treatments. Zilucoplan and pegcetacoplan, for example, are designed to block the complement pathway earlier in the cascade, providing broader inhibition that may benefit patients with partial or inadequate responses to traditional C5 inhibitors. These alternative pathway inhibitors aim to prevent hemolysis more effectively and reduce the need for blood transfusions, offering hope for patients who do not achieve full remission with current therapies. As these new inhibitors reach advanced clinical trials, they hold potential to expand PNH treatment options, providing a solution for those who require more comprehensive complement inhibition.

Gene therapy and CRISPR-based research are also advancing as potential long-term solutions for PNH, with the goal of correcting the genetic defect that causes the disease. Although still in early stages of research, gene therapy aims to provide a one-time treatment that can potentially cure or significantly reduce the need for ongoing medication. By directly targeting the genetic mutation that leads to PNH, gene therapy could offer a permanent solution for patients. While these technologies are still under development, their potential for long-lasting efficacy makes them an exciting area of research. As gene therapy advances, it may revolutionize PNH treatment, offering patients hope for a future without lifelong reliance on medication.

What Are the Benefits of PNH Treatments for Patients and Healthcare Systems?

PNH treatments provide significant benefits for patients by managing symptoms, reducing the risk of life-threatening complications, and improving quality of life, while healthcare systems benefit from reduced hospitalization and improved patient outcomes. For patients, complement inhibitors like eculizumab and ravulizumab have transformed PNH management by reducing hemolysis, which alleviates symptoms such as fatigue, weakness, and difficulty breathing. By reducing hemolysis, these treatments help patients maintain healthier hemoglobin levels, enhancing their ability to participate in daily activities. For patients with PNH, effective symptom management is critical for maintaining independence and reducing the impact of the disease on their daily lives.

Complement inhibitors also play a crucial role in reducing the risk of thrombosis, which is one of the leading causes of mortality in PNH patients. By preventing clot formation, these treatments decrease the likelihood of severe complications such as strokes, pulmonary embolisms, and organ damage. This reduction in thrombotic events is particularly beneficial for patients’ long-term survival, as untreated thrombosis can lead to fatal outcomes. Effective treatment with complement inhibitors thus reduces the need for emergency interventions, improving the stability of patients' health and providing a better long-term prognosis.

For healthcare systems, PNH treatments reduce the need for frequent hospitalizations and emergency care, lowering overall healthcare costs associated with managing complications. As treatments like ravulizumab allow for less frequent dosing, they decrease the frequency of hospital visits and infusion sessions, freeing up healthcare resources and improving cost efficiency. By providing effective, long-acting solutions, healthcare systems can focus on preventive care rather than acute interventions, which supports better patient outcomes. Furthermore, as new treatments improve the effectiveness of PNH management, patients are less reliant on supportive care like blood transfusions and anticoagulants, which further reduces healthcare expenses and resource allocation.

Additionally, the development of oral complement inhibitors in clinical trials could offer even greater convenience and access to treatment for patients, especially in regions where access to infusion centers is limited. Oral therapies would allow patients to manage their condition from home, eliminating the need for frequent travel to healthcare facilities. This accessibility can greatly benefit rural or underserved populations, providing them with the necessary treatment without the logistical and financial burden of travel. For healthcare systems, oral therapies would reduce the demand on infusion centers and hospital resources, further supporting efficient care delivery and improving accessibility for all PNH patients.

What Is Fueling the Growth in the PNH Treatment Market?

The growth in the PNH treatment market is driven by increased awareness of rare diseases, advancements in targeted therapies, expanded patient access to new treatments, and the emergence of gene therapy research. With greater awareness and understanding of PNH among healthcare providers and patients, diagnosis rates have improved, leading to earlier and more accurate treatment initiation. As patients are diagnosed sooner, they can begin receiving life-saving treatments that reduce complications and improve long-term outcomes. Awareness efforts, often driven by patient advocacy groups, have also encouraged more investment in PNH research, leading to the development of advanced therapies and expanded treatment options. As awareness and diagnosis improve, the demand for effective PNH treatments continues to grow.

Advancements in targeted therapies, particularly in complement inhibitors, are a major growth driver, as these treatments offer effective control of hemolysis and reduce the risk of thrombotic events. The success of C5 inhibitors like eculizumab and ravulizumab has paved the way for the development of next-generation therapies, including C3 inhibitors and other complement pathway inhibitors. These new therapies are expanding treatment options and addressing unmet needs for patients with incomplete responses to existing treatments. The market’s growth is further supported by increasing interest in rare disease research, which has encouraged pharmaceutical companies to invest in innovative treatments for PNH. As companies pursue novel therapies and expand the range of complement inhibitors, the PNH treatment market is set to grow.

Expanded patient access to PNH treatments, facilitated by regulatory approvals and broader insurance coverage, is also driving market growth. Regulatory agencies such as the FDA and EMA have granted orphan drug status to many PNH treatments, streamlining approval processes and increasing availability in different regions. Insurance coverage for high-cost therapies like complement inhibitors has also improved, making these treatments more accessible to patients who previously may not have been able to afford them. Additionally, the introduction of long-acting therapies, which require less frequent dosing, has made treatment more convenient and accessible for patients, particularly those in remote or underserved areas. As access to PNH treatments expands globally, the market is expected to grow, reaching more patients and improving outcomes across diverse regions.

The emergence of gene therapy research holds promise for transforming the PNH treatment market in the long term. By addressing the underlying genetic mutation that causes PNH, gene therapy has the potential to offer a one-time, curative treatment that eliminates the need for lifelong medication. Although still in early stages, gene therapy research has garnered significant interest due to its potential to provide long-lasting relief from PNH symptoms and complications. Investment in gene therapy and CRISPR-based solutions has increased as companies explore their application in PNH treatment. Should these therapies prove successful, they would revolutionize the PNH market, offering a permanent solution for patients and reducing reliance on costly, ongoing treatments. Together, these factors—greater awareness, targeted therapeutic advancements, increased patient access, and gene therapy research—are driving robust growth in the PNH treatment market, establishing it as a key area in rare disease management and innovative healthcare.

SCOPE OF STUDY:

The report analyzes the Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment market in terms of US$ Million by the following Treatment, and Geographic Regions/Countries:

Segments:

Treatment (Medication, Other Treatments)

Geographic Regions/Countries:

World; United States; Canada; Japan; China; Europe (France; Germany; Italy; United Kingdom; and Rest of Europe); Asia-Pacific; Rest of World.

Select Competitors (Total 42 Featured) -

• Akari Therapeutics Plc
• Alexion Pharmaceuticals, Inc.
• Apellis Pharmaceuticals
• AstraZeneca Plc
• CANbridge Pharmaceuticals
• Chugai Pharmaceutical Co., Ltd.
• F. Hoffmann-La Roche AG
• Genentech, Inc.
• Kira Pharmaceuticals
• Novartis AG

TABLE OF CONTENTS

I. METHODOLOGY

II. EXECUTIVE SUMMARY

• 1. MARKET OVERVIEW
  • Influencer Market Insights
  • World Market Trajectories
  • Global Economic Update
  • Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment - Global Key Competitors Percentage Market Share in 2024 (E)
  • Competitive Market Presence - Strong/Active/Niche/Trivial for Players Worldwide in 2024 (E)
• 2. FOCUS ON SELECT PLAYERS
• 3. MARKET TRENDS & DRIVERS
  • Rising Prevalence of PNH Drives Demand for Novel Therapeutics
  • Advancements in Gene Therapy Drive PNH Treatment Adoption
  • Growing Use of Monoclonal Antibodies in PNH Treatment Expands Market Potential
  • Increasing Adoption of Hematopoietic Stem Cell Transplantation Spurs Demand
  • Rising Awareness Initiatives Enhance Early Diagnosis of PNH
  • Innovations in Eculizumab Alternatives Propel PNH Treatment Market
  • Expanding Applications of Complement Blockers Set the Stage for PNH Growth
  • Use of Advanced Diagnostic Tools Bodes Well for PNH Treatment Expansion
  • Growing Use of Biosimilars in PNH Treatment Expands Addressable Market
  • Demand for Supportive Therapies in PNH Treatment Propels Market Growth
• 4. GLOBAL MARKET PERSPECTIVE
  • TABLE 1: World Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2023 through 2030 and % CAGR
  • TABLE 2: World Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
  • TABLE 3: World 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets for Years 2014, 2024 & 2030
  • TABLE 4: World Recent Past, Current & Future Analysis for Medication by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2023 through 2030 and % CAGR
  • TABLE 5: World Historic Review for Medication by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
  • TABLE 6: World 16-Year Perspective for Medication by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2014, 2024 & 2030
  • TABLE 7: World Recent Past, Current & Future Analysis for Other Treatments by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2023 through 2030 and % CAGR
  • TABLE 8: World Historic Review for Other Treatments by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
  • TABLE 9: World 16-Year Perspective for Other Treatments by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2014, 2024 & 2030
  • TABLE 10: World Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Analysis of Annual Sales in US$ Million for Years 2014 through 2030

III. MARKET ANALYSIS

• UNITED STATES
  • Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in the United States for 2024 (E)
  • TABLE 11: USA Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
  • TABLE 12: USA Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
  • TABLE 13: USA 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
• CANADA
  • TABLE 14: Canada Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
  • TABLE 15: Canada Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
  • TABLE 16: Canada 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
• JAPAN
  • Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Japan for 2024 (E)
  • TABLE 17: Japan Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
  • TABLE 18: Japan Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
  • TABLE 19: Japan 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
• CHINA
  • Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in China for 2024 (E)
  • TABLE 20: China Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
  • TABLE 21: China Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
  • TABLE 22: China 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
• EUROPE
  • Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Europe for 2024 (E)
  • TABLE 23: Europe Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Million for Years 2023 through 2030 and % CAGR
  • TABLE 24: Europe Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
  • TABLE 25: Europe 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Geographic Region - Percentage Breakdown of Value Sales for France, Germany, Italy, UK and Rest of Europe Markets for Years 2014, 2024 & 2030
  • TABLE 26: Europe Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
  • TABLE 27: Europe Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
  • TABLE 28: Europe 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
• FRANCE
  • Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in France for 2024 (E)
  • TABLE 29: France Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
  • TABLE 30: France Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
  • TABLE 31: France 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
• GERMANY
  • Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Germany for 2024 (E)
  • TABLE 32: Germany Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
  • TABLE 33: Germany Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
  • TABLE 34: Germany 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
• ITALY
  • TABLE 35: Italy Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
  • TABLE 36: Italy Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
  • TABLE 37: Italy 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
• UNITED KINGDOM
  • Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in the United Kingdom for 2024 (E)
  • TABLE 38: UK Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
  • TABLE 39: UK Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
  • TABLE 40: UK 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
• REST OF EUROPE
  • TABLE 41: Rest of Europe Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
  • TABLE 42: Rest of Europe Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
  • TABLE 43: Rest of Europe 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
• ASIA-PACIFIC
  • Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Asia-Pacific for 2024 (E)
  • TABLE 44: Asia-Pacific Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
  • TABLE 45: Asia-Pacific Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
  • TABLE 46: Asia-Pacific 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030
• REST OF WORLD
  • TABLE 47: Rest of World Recent Past, Current & Future Analysis for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments - Independent Analysis of Annual Sales in US$ Million for the Years 2023 through 2030 and % CAGR
  • TABLE 48: Rest of World Historic Review for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Medication and Other Treatments Markets - Independent Analysis of Annual Sales in US$ Million for Years 2014 through 2022 and % CAGR
  • TABLE 49: Rest of World 16-Year Perspective for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment by Treatment - Percentage Breakdown of Value Sales for Medication and Other Treatments for the Years 2014, 2024 & 2030

IV. COMPETITION