Results from the first four regimens of the HEALEY ALS Platform Trial (PL5.004)

Paganoni, Sabrina; Berry, James; Quintana, Melanie; Macklin, Eric A.; Saville, Benjamin R.; Andrews, Jinsy; Shefner, Jeremy M.; Fournier, Christina; Babu, Suma; Maragakis, Nicholas J.; Oskarsson, Björn; Detry, Michelle A.; Chase, Marianne; Sherman, Alex; Yu, Hong; Pothier, Lindsay; Drake, Kristin; Chibnik, Lori B.; Bind, Marie-Abèle; Vestrucci, Matteo; McGlothlin, Anna; Marion, Joseph; Duda, Petra W.; Harvey, Brittany; Qureshi, Irfan A.; Donohue, Mary; Granit, Volkan; Grossman, Katheryn; Glanzman, Robert; Hotchkin, Michael; Goldberg, Y P; Leitner, Melanie; Hayden, Michael R.; Cudkowicz, Merit

doi:10.1212/wnl.0000000000204032

Cited by 2 publications

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“…In addition to the approvals in Japan, the USA and the EU, zilucoplan is also currently under regulatory review in Australia and Canada for use in the treatment of gMG. Clinical development of zilucoplan in other indications, including paroxysmal nocturnal haemoglobinuria [ 7 ], immune-mediated necrotising myopathy [ 8 ], amyotrophic lateral sclerosis [ 9 ] and patients hospitalised for COVID-19 [ 10 – 12 ], has been discontinued.…”

Section: Introductionmentioning

confidence: 99%

Zilucoplan: First Approval

Shirley

2023

Drugs

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Zilucoplan (Zilbrysq ® ) is a subcutaneously administered macrocyclic peptide inhibitor of complement component 5 (C5 inhibitor) being developed by UCB for the treatment of generalised myasthenia gravis (gMG). Zilucoplan received its first approval, in Japan, in September 2023 for the treatment of gMG in adult patients who inadequately respond to steroids or other immunosuppressants and are positive for anti-acetylcholine receptor (AChR) antibodies. Subsequently, zilucoplan was approved in the USA in October 2023 for the treatment of gMG in adult patients who are anti-AChR antibody positive and in the EU in December 2023 as an add-on to standard therapy for the treatment of gMG in adult patients who are anti-AChR antibody positive. Zilucoplan is also currently under regulatory review in Australia and Canada for use in the treatment of gMG. This article summarises the milestones in the development of zilucoplan leading to this first approval for gMG. Supplementary Information The online version contains supplementary material available at 10.1007/s40265-023-01977-3.

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Section: Introductionmentioning

confidence: 99%