Complement in human disease: approved and up-and-coming therapeutics

EE West, T Woodruff, V Fremeaux-Bacchi, C Kemper - The Lancet, 2024 - thelancet.com
The complement system is recognised as a protector against blood-borne pathogens and a
controller of immune system and tissue homoeostasis. However, dysregulated complement …

Therapeutic targeting of the complement system: from rare diseases to pandemics

P Garred, AJ Tenner, TE Mollnes, FO Levy - Pharmacological reviews, 2021 - Elsevier
The complement system was discovered at the end of the 19th century as a heat-labile
plasma component that “complemented” the antibodies in killing microbes, hence the name …

Pegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria

P Hillmen, J Szer, I Weitz, A Röth… - … England Journal of …, 2021 - Mass Medical Soc
Background Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired disease
characterized by chronic complement-mediated hemolysis. C5 inhibition controls …

Oral iptacopan monotherapy in paroxysmal nocturnal hemoglobinuria

R Peffault de Latour, A Röth… - … England Journal of …, 2024 - Mass Medical Soc
Background Persistent hemolytic anemia and a lack of oral treatments are challenges for
patients with paroxysmal nocturnal hemoglobinuria who have received anti-C5 therapy or …

Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor–experienced adult patients with PNH: the 302 study

AG Kulasekararaj, A Hill, ST Rottinghaus… - Blood, The Journal …, 2019 - ashpublications.org
Ravulizumab, a new complement component C5 inhibitor administered every 8 weeks, was
noninferior to eculizumab administered every 2 weeks in complement-inhibitor–naive …

Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study

JW Lee, F Sicre de Fontbrune… - Blood, The Journal …, 2019 - ashpublications.org
Abstract Ravulizumab (ALXN1210), a new complement C5 inhibitor, provides immediate,
complete, and sustained C5 inhibition. This phase 3, open-label study assessed the …

Anti-complement treatment for paroxysmal nocturnal hemoglobinuria: time for proximal complement inhibition? A position paper from the SAAWP of the EBMT

AM Risitano, S Marotta, P Ricci, L Marano… - Frontiers in …, 2019 - frontiersin.org
The treatment of paroxysmal nocturnal hemoglobinuria has been revolutionized by the
introduction of the anti-C5 agent eculizumab; however, eculizumab is not the cure for …

Small-molecule factor B inhibitor for the treatment of complement-mediated diseases

A Schubart, K Anderson, N Mainolfi… - Proceedings of the …, 2019 - National Acad Sciences
Dysregulation of the alternative complement pathway (AP) predisposes individuals to a
number of diseases including paroxysmal nocturnal hemoglobinuria, atypical hemolytic …

Eculizumab in paroxysmal nocturnal haemoglobinuria and atypical haemolytic uraemic syndrome: 10‐year pharmacovigilance analysis

G Socié, MP Caby‐Tosi, JL Marantz… - British journal of …, 2019 - Wiley Online Library
Eculizumab is the first and only medication approved for paroxysmal nocturnal
haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) treatment …

Addition of iptacopan, an oral factor B inhibitor, to eculizumab in patients with paroxysmal nocturnal haemoglobinuria and active haemolysis: an open-label, single …

AM Risitano, A Röth, J Soret, C Frieri… - The Lancet …, 2021 - thelancet.com
Background The haematological benefit of standard-of-care anti-C5 treatment for haemolytic
paroxysmal nocturnal haemoglobinuria is limited by residual intravascular haemolysis or …