Paroxysmal Nocturnal Hemoglobinuria – Landscape & Forecast – Niche & Rare Disease Landscape & Forecast (US/EU5)

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder in which the immune system attacks red blood cells and platelets. PNH is characterized by intravascular hemolysis, venous thrombosis, and cytopenia associated with bone marrow failure. Patients present with fatigue, dyspnea, renal insufficiency, and dark urine due to marked hemoglobinuria. For a long time, treatment was only supportive, consisting of blood transfusions, iron supplementation, and antithrombotic agents. In the last decade, the approval of injectable complement inhibitors such as Alexion’s eculizumab (Soliris) and ravulizumab (Ultomiris) and Apellis Pharmaceuticals’ pegcetacoplan (Empaveli) has transformed the treatment landscape, offering PNH patients a better quality of life and improved survival. Novartis’s iptacopan (Fabhalta) is the first oral therapy approved by the FDA and EC for PNH, followed by AstraZeneca’s danicopan (Voydeya). However, all approved complement inhibitors carry the risk of life-threatening infections / sepsis. Thus, safer and more-effective treatments are needed.

Questions answered

• How big is the PNH patient population in the United States and EU5, and how will the population change through 2034?
• How will the launch of Fabhalta and Voydeya impact the PNH therapy market?
• What are the biggest unmet needs in the treatment of PNH?
• Which emerging therapies will launch before 2034, and what will be their commercial impact on the PNH market through 2034?

Geographies: United States and EU5.

Primary research: Country-specific interviews with thought-leading hematologists. Survey data collected for this and other Clarivate research.

Epidemiology: Diagnosed prevalent and drug-treated cases of PNH by country.

Forecast: Drug-level sales and patient share of key PNH therapies in 2024 and 2034.

Drug treatments: Coverage of key current and late-phase emerging therapies.

Product description: Niche & Rare Disease Landscape & Forecast offers comprehensive market intelligence with world-class epidemiology, keen insight into current and emerging therapies, and drug forecasts supported by detailed primary and secondary research, enabling you to:

• Optimize your long-term disease and development strategy.
• Quantify market potential for your pipeline assets and those of your competitors.
• Understand a disease from top to bottom, including key patient populations, the current and future therapeutic landscape, and the evolving market trajectory.
• Gauge the commercial outlook and impact of key market events.