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Innovation Observatory > Reports > Drugs > Pegcetacoplan (APL-2) for paroxysmal nocturnal haemoglobinuria

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Pegcetacoplan (APL-2) for paroxysmal nocturnal haemoglobinuria


December 2019

Pegcetacoplan (APL-2) is a medicinal product in clinical development for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH). PNH is an ultra‐rare life‐threatening blood disorder. PNH leads to excessive breakdown of red blood cells, leading to the release of a large amount of haemoglobin (the protein found in red blood cells that carries oxygen around the body) into the urine. Symptoms and signs of PNH are varied and can include: fatigue; dark red/brown urine; difficulty swallowing, abdominal pain, infections, and bruising. PNH typically starts from the early thirties to the mid‐forties, and often persisting for decades, with a continued dependence on blood transfusions in a proportion of patients.
Pegcetacoplan targets and inhibits the activation of an important protein in the complement system called C3 that plays a key role in PNH. By inhibiting C3 activation, the medicine is expected to block the chain of reactions that damage the red blood cells, thus relieving the symptoms of the disease. Pegcetacoplan is given by subcutaneous injection for self-administration. If licensed, Pegcetacoplan could represent an additional treatment option for patients with PNH.

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