2024年11月5日 · In December 2023, iptacopan was approved as the first orally administered monotherapy for patients with PNH in the United States (US). Since then, two more CIs have been approved (danicopan and crovalimab). With an increasing number of CIs available for PNH, timely real-world evidence is needed to inform treatment decision-making.

2023年7月31日 · For instance, about 30 to 40% of PNH cases are reported in the United States(US) and Europe, whereas less than 10% of PNH cases are reported from Asia. Consequently, the incidence of thromboembolism due to PNH is higher in the US and Europe compared to Japan.

2023年11月2日 · PNH is clinically heterogeneous, and hospitalization and regular blood transfusions may be needed. Treatment with currently approved C5 inhibitors (C5i), eculizumab and ravulizumab, have shown efficacy in intravascular hemolysis control …

2019年11月13日 · The objective of this study was to estimate the incidence and prevalence of PNH and to describe real-world treatment patterns among patients newly diagnosed with PNH in the United States (US).

In the United States, researchers estimate the prevalence of monoclonal PNH cases at 3000 individuals and biclonal (or multiclonal) PNH cases at 2 individuals based on the 2010 US Census Bureau. Other estimations for PNH incidence and prevalence in the United States are based on research conducted in the United Kingdom. 7

To date, the only therapies for PNH approved by the U.S. Food and Drug Administration (FDA) are the complement-inhibitory drugs eculizumab (approved in 2007) and ravulizumab (approved in 2018), both monoclonal antibodies designed to target the complement protein C5, thereby preventing its cleavage and the formation of the terminal attack complex...

Patients with paroxysmal nocturnal hemoglobinuria (PNH) often experience a lengthy path to diagnosis. Fewer than 40% of patients with PNH receive a diagnosis within 12 months of symptom onset, and 24% of all PNH diagnoses can take 5 years or longer. Diagnostic delay is a source of distress and can affect emotional well-being for patients with PNH.

2024年4月15日 · NM5072 is being developed for PNH patients in the United States and globally. The FDA grants ODD status to new medication intended for the treatment, diagnosis or prevention of rare...

The objective of this study was to determine C5i treatment effects on clinical parameters, PNH symptoms, quality of life, and resource use for PNH patients. This cross-sectional study surveyed 122 individuals in the USA receiving treatment for PNH with C5-targeted monoclonal antibodies, eculizumab (ECU) or ravulizumab (RAV).

2024年11月5日 · This interim analysis aims to provide early insights into the characteristics and treatment patterns of patients enrolled in APPRISE-PNH to date. Methods: APPRISE-PNH is a retrospective, longitudinal, data platform including adult patients (age ≥18 years) with PNH treated with iptacopan in the US real-world setting. Consenting patients in the ...