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1595057

세계의 발작성야간혈색뇨증(PNH) 치료 시장

Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment

발행일: | 리서치사: Market Glass, Inc. (Formerly Global Industry Analysts, Inc.) | 페이지 정보: 영문 93 Pages | 배송안내 : 1-2일 (영업일 기준)

    
    
    



※ 본 상품은 영문 자료로 한글과 영문 목차에 불일치하는 내용이 있을 경우 영문을 우선합니다. 정확한 검토를 위해 영문 목차를 참고해주시기 바랍니다.

세계의 발작성야간혈색뇨증(PNH) 치료 시장은 2030년까지 84억 달러에 도달

2023년에 41억 달러로 추정되는 세계의 발작성야간혈색뇨증(PNH) 치료 시장은 2023-2030년의 분석 기간에 CAGR 10.7%로 성장하며, 2030년에는 84억 달러에 달할 것으로 예측됩니다. 본 리포트에서 분석한 부문의 하나인 투약 치료는 CAGR 9.0%를 기록하며, 분석 기간 종료시에는 55억 달러에 달할 것으로 예측됩니다. 기타 치료 분야의 성장률은 분석 기간에 CAGR 14.4%로 추정됩니다.

미국 시장은 11억 달러로 추정, 중국은 CAGR 10.1%로 성장 예측

미국의 발작성야간혈색뇨증(PNH) 치료 시장은 2023년에 11억 달러로 추정됩니다. 세계 2위의 경제대국인 중국은 2030년까지 13억 달러의 시장 규모에 달할 것으로 예측되며, 분석 기간인 2023-2030년의 CAGR은 10.1%입니다. 기타 주목해야 할 지역별 시장으로는 일본과 캐나다가 있으며, 분석 기간 중 CAGR은 각각 9.1%와 8.9%로 예측됩니다. 유럽에서는 독일이 CAGR 7.8%로 성장할 것으로 예측됩니다.

세계의 발작성야간혈색뇨증(PNH) 치료 시장 - 주요 동향과 촉진요인 정리

발작성야간혈색뇨증(PNH)의 효과적 치료가 필수적인 이유는?

발작성야간혈색뇨증(PNH)의 효과적인 치료는 이 희귀한 혈액 질환에 수반되는 심각하고 생명을 위협하는 합병증으로 인해 필수적이며, PNH는 적혈구가 조기에 떨어져 나가 용혈성 빈혈과 혈전증을 유발하고 심할 경우 장기 손상과 사망에 이르게 합니다. 이 질환은 면역 매개 적혈구 파괴를 동반하므로 환자는 항상 피로, 복통, 호흡곤란, 혈전과 같은 쇠약 증상의 위험에 노출되어 있습니다. 치료하지 않으면 이러한 합병증은 환자의 삶의 질에 심각한 영향을 미치고 뇌졸중이나 장기 부전과 같은 생명을 위협하는 상황을 초래할 수 있으며, PNH의 치료는 용혈 예방, 혈전 사건 감소 및 증상 관리에 중점을 두어 환자의 결과를 개선하는 데 초점을 맞추었습니다.

PNH의 치료는 일반적으로 면역계를 표적으로 하는 치료, 특히 면역계가 적혈구를 공격하는 것을 막는 보체 억제제를 사용합니다. 에크리주맙(솔리리스)이나 라브릴주맙(울트라마브)과 같은 보체 억제제의 도입은 용혈률을 낮추고 혈전증 위험을 감소시킴으로써 PNH 관리에 변화를 가져왔으며, 환자들에게 더 긴 수명과 더 나은 삶의 질을 제공했습니다. 혈전증, 특히 정맥혈전증의 위험이 높은 PNH에서 보체 억제제는 이러한 합병증을 줄이고 환자가 보다 안정적인 삶을 영위할 수 있도록 하는 데 매우 중요한 역할을 합니다. 중요합니다.

보체 억제제 외에도 빈혈을 관리하기 위한 수혈과 혈전 형성을 예방하기 위한 약수처리와 같은 지지요법이 PNH의 관리에 중요한 역할을 합니다. 수혈은 헤모글로빈 농도를 유지하고 피로감, 쇠약감 등 빈혈 관련 증상에 대처하는 데 도움이 되며, 항응고제는 혈전 위험을 감소시키는 데 도움이 됩니다. 다각적인 접근이 필요하며, 효과적인 치료는 환자를 안정시키고, 합병증을 예방하며, 궁극적으로 PNH 환자의 삶의 질을 향상시키는 데 필수적입니다.

기술적, 약리학적인 발전은 PNH 치료를 어떻게 변화시킬 것인가?

기술적, 약리학적인 발전은 더 오래 지속되는 효과, 개선된 안전성, 환자 편의성을 갖춘 보다 효과적인 치료법을 제공함으로써 PNH 치료를 변화시키고 있으며, PNH 치료의 가장 중요한 혁신 중 하나는 에클리주맙과 같은 초기 치료제보다 투여 횟수를 줄일 수 있는 라브리주맙과 같은 장기 지속형 보체 억제제의 개발입니다. 투여 횟수를 줄일 수 있는 라브릴주맙과 같은 장시간 작용하는 보체 억제제의 개발입니다. 라브릴주맙은 투약 간격을 2주 간격에서 8주 간격으로 연장하여 환자에게 편의성을 제공하고 치료 순응도를 향상시킵니다. 이러한 투여 일정 연장은 잦은 병원 방문이 어렵고 시간이 많이 걸리는 환자들에게 특히 유용합니다. 잦은 투약의 부담을 줄임으로써 장시간 작용하는 보체 억제제는 보다 관리하기 쉬운 치료 루틴을 지원하여 보다 환자 친화적인 치료 경험을 제공합니다.

C3 억제제 및 대체 경로 억제제와 같은 대체 경로를 표적으로 하는 새로운 보체 억제제는 기존 치료제에 내성을 보이는 환자들에게 유망한 대안으로 떠오르고 있습니다. 예를 들어 지르코플란과 페그세타코플란은 보체 경로의 초기 단계에서 보체 경로를 차단하도록 설계되어 기존 C5 억제제에 부분적 또는 불충분한 반응을 보이는 환자들에게 보다 광범위한 억제 효과가 있으며, 도움이 될 수 있습니다. 이러한 대체 경로 억제제는 용혈을 보다 효과적으로 예방하고 수혈의 필요성을 줄이는 것을 목표로 하고 있으며, 현재 치료법으로 완전한 관해에 도달하지 못한 환자들에게 희망을 주고 있습니다. 이러한 새로운 억제제가 임상시험 단계에 도달하면 PNH 치료 옵션을 확장하고보다 종합적인 보체 억제가 필요한 환자에게 해결책을 제공할 수 있습니다.

유전자 치료와 CRISPR에 기반한 연구도 PNH의 장기적인 해결책이 될 수 있는 연구로 발전하고 있습니다. 아직 연구 초기 단계에 있지만, 유전자 치료는 일회성 치료로 완치하거나 지속적인 약물의 필요성을 크게 줄이는 것을 목표로 하고 있으며, PNH를 유발하는 유전자 돌연변이를 직접 표적으로 삼음으로써 유전자 치료는 환자에게 영구적인 해결책을 제공할 수 있습니다. 이러한 기술은 아직 개발 중이지만, 장기적인 효과를 기대할 수 있으므로 흥미로운 연구 분야입니다. 유전자 치료가 발전하면 PNH 치료에 혁명을 일으키고 환자들에게 평생 약물에 의존하지 않는 미래에 대한 희망을 가져다 줄 수 있습니다.

PNH 치료는 환자와 의료 시스템에 어떤 이점이 있는가?

PNH 치료는 증상 관리, 생명을 위협하는 합병증 위험 감소, 삶의 질 개선 등 환자들에게 큰 이점을 제공하며, 의료 시스템에는 입원 감소, 환자 예후 개선 등 다양한 이점을 제공합니다. 환자의 경우, 에크리주맙과 라브릴주맙과 같은 보체 억제제는 용혈을 감소시키고 피로, 쇠약, 호흡곤란과 같은 증상을 완화함으로써 PNH 관리에 큰 변화를 가져왔습니다. 용혈을 감소시킴으로써 이러한 치료는 환자가 더 건강한 헤모글로빈 농도를 유지하고 일상 활동에 참여하는 능력을 향상시키는 데 도움이되며, PNH 환자의 경우 효과적인 증상 관리는 독립성을 유지하고 일상 생활에 미치는 영향을 줄이기 위해 매우 중요합니다.

보체 억제제는 또한 PNH 환자의 주요 사망 원인 중 하나인 혈전증의 위험을 줄이는 데 중요한 역할을 합니다. 혈전 형성을 예방함으로써 이러한 치료는 뇌졸중, 폐색전증, 장기 손상과 같은 심각한 합병증의 가능성을 감소시킵니다. 치료되지 않은 혈전증은 치명적인 결과를 초래할 수 있으므로 이러한 혈전 사건의 감소는 환자의 장기 생존에 특히 유익합니다. 따라서 보체 억제제를 통한 효과적인 치료는 응급 개입의 필요성을 줄이고 환자의 건강 상태를 안정화하며 장기 예후를 개선합니다.

보건의료 시스템에서 PNH 치료는 잦은 입원 및 응급 치료의 필요성을 줄여 합병증 관리와 관련된 전체 의료 비용을 절감하고, 라불리주맙과 같은 치료제는 투여 횟수를 줄여 병원 방문과 정맥주사 횟수를 줄여 의료 자원을 확보하고 비용 효율을 높입니다. 비용 효율성이 향상됩니다. 효과적이고 오래 지속되는 솔루션을 제공함으로써 의료 시스템은 급성기 개입보다 예방 의료에 집중할 수 있으며, 환자의 예후를 개선할 수 있습니다. 또한 새로운 치료법이 PNH 관리의 효과를 개선함에 따라 환자는 수혈이나 항응고제와 같은 지지요법에 덜 의존하게 되어 의료 비용과 자원 배분을 더욱 절감할 수 있습니다.

또한 임상시험에서 경구용 보체 억제제가 개발되면, 특히 수액 센터에 대한 접근성이 제한된 지역에서 환자들의 치료 편의성과 접근성을 더욱 향상시킬 수 있습니다. 경구용 치료제를 통해 환자들은 집에서 병을 관리할 수 있게 되고, 의료기관을 자주 방문하지 않아도 됩니다. 이러한 접근성은 농촌 지역이나 의료 혜택이 부족한 사람들에게는 이동에 따른 물류 및 경제적 부담 없이 필요한 치료를 받을 수 있다는 큰 이점이 있습니다. 의료시스템의 경우, 경구용 치료는 수액센터와 병원의 자원에 대한 수요를 줄여 효율적인 치료 제공을 더욱 지원하고 모든 PNH 환자의 접근성을 향상시킬 수 있습니다.

PNH 치료 시장의 성장을 가속하는 요인은 무엇인가?

희귀질환에 대한 인식 증가, 표적 치료의 발전, 새로운 치료법에 대한 환자 접근성 확대, 유전자 치료 연구의 부상 등이 PNH 치료 시장의 성장 동력이 되고 있습니다. 의료진과 환자들 사이에서 PNH에 대한 인식과 이해가 높아짐에 따라 진단율이 향상되고 정확한 치료를 더 빨리 시작할 수 있게 되었습니다. 환자가 더 빨리 진단을 받으면 합병증을 줄이고 장기적인 결과를 개선할 수 있는 생명을 구하는 치료를 받을 수 있습니다. 또한 환자 지원 단체의 인식 개선 활동은 PNH 연구에 대한 투자를 촉진하여 첨단 치료법 개발과 치료 옵션의 확대로 이어지고 있으며, PNH에 대한 인식과 진단이 향상됨에 따라 효과적인 PNH 치료에 대한 수요는 지속적으로 증가하고 있습니다.

표적 치료, 특히 보체 억제제의 발전은 용혈을 효과적으로 제어하고 혈전 사건의 위험을 줄일 수 있으므로 큰 촉진제 역할을 하고 있습니다. 에클리주맙과 라브리주맙과 같은 C5 억제제의 성공은 C3 억제제 및 기타 보체 경로 억제제를 포함한 차세대 치료제 개발의 길을 열었습니다. 이러한 새로운 치료제는 치료 선택의 폭을 넓히고 기존 치료제로는 효과가 충분하지 않은 환자의 미충족 수요를 충족시킬 수 있습니다. 희귀질환 연구에 대한 관심이 높아지면서 제약사들이 PNH에 대한 혁신적인 치료법에 대한 투자를 늘리면서 시장 성장에 더욱 박차를 가하고 있습니다. 제약사들이 새로운 치료법을 추구하고 보체 억제제의 유형을 늘리면서 PNH 치료 시장은 성장할 것으로 보입니다.

규제 당국의 승인과 보험 적용 확대에 힘입어 PNH 치료제에 대한 환자 접근성 확대도 시장 성장의 원동력이 되고 있으며, FDA 및 EMA와 같은 규제기관은 많은 PNH 치료제에 희귀의약품(Orphan Drug) 지위를 부여하여 승인 절차를 간소화하고 다양한 지역에서의 접근성을 높이고 있습니다. 승인 절차를 간소화하고 다양한 지역에서의 접근성을 높이고 있습니다. 보체 억제제와 같은 고가의 치료제에 대한 보험 적용도 개선되어 이전에는 치료비를 지불할 수 없었던 환자들이 치료제를 더 쉽게 이용할 수 있게 되었습니다. 또한 더 적은 횟수로 투여할 수 있는 지속형 치료제의 도입으로, 특히 외딴 지역이나 치료 접근성이 낮은 지역의 환자들에게 더 편리하고 쉽게 치료받을 수 있게 되었습니다. 다양한 지역에서 치료 결과가 개선될 것으로 예상됩니다.

유전자 치료 연구의 출현은 장기적으로 PNH 치료 시장을 변화시킬 수 있는 잠재력을 가지고 있으며, PNH를 유발하는 근본적인 유전자 돌연변이를 해결함으로써 유전자 치료는 평생 투약의 필요성을 없애고 단 한 번의 치료로 완치할 수 있는 치료법을 제공할 수 있는 잠재력을 가지고 있습니다. 유전자 치료 연구는 아직 초기 단계에 있지만, PNH의 증상과 합병증을 장기적으로 완화할 수 있다는 점에서 큰 관심을 받고 있습니다. 기업이 PNH 치료에 적용을 모색하면서 유전자 치료와 CRISPR 기반 솔루션에 대한 투자가 증가하고 있습니다. 이러한 치료법이 성공한다면 PNH 시장에 혁명을 일으켜 환자에게 영구적인 해결책을 제공하고 비용이 많이 드는 지속적인 치료에 대한 의존도를 낮출 수 있습니다. 인지도의 향상, 표적 치료의 발전, 환자 접근성 향상, 유전자 치료 연구 등 이러한 요인들이 결합하여 PNH 치료 시장은 강력한 성장을 이루며 희귀질환 관리 및 혁신적 헬스케어의 중요한 분야로 자리매김하고 있습니다.

부문

치료(약물 요법, 기타 치료)

조사 대상 기업의 예(총 42건)

  • 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

목차

제1장 조사 방법

제2장 개요

  • 시장 개요
  • 주요 기업
  • 시장 동향과 촉진요인
  • 세계 시장의 전망

제3장 시장 분석

  • 미국
  • 캐나다
  • 일본
  • 중국
  • 유럽
  • 프랑스
  • 독일
  • 이탈리아
  • 영국
  • 기타 유럽
  • 아시아태평양
  • 기타 지역

제4장 경쟁

KSA 24.11.27

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

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