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 for PNH. 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 countries, and how will the population change through 2033?
• How will the launch of recently approved Fabhalta impact the PNH therapy market?
• What are the biggest unmet needs in the treatment of PNH?
• Which emerging therapies will launch before 2033, and what will be their commercial impact on the PNH market through 2033?

Geographies: United States and EU5.

Primary research: Six country-specific interviews with thought-leading hematologists. Supported by survey data collected for this study.

Epidemiology: Diagnosed prevalent cases of PNH by country.

Forecast: Drug-level sales and patient share of key PNH therapies through 2033.

Drug treatments: Coverage of key current and emerging therapies.

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

Key feature: Niche & Rare Disease Landscape & Forecast features a Drug Pipeline chapter with real-time, global pipeline intelligence content directly from Cortellis. This chapter is updated daily and features interactive figures that can be easily downloaded for detailed analysis or presentations.