MHRA provides Marketing Authorisation for Pegcetacoplan (Aspaveli)

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Following a Great Britain Marketing Authorisation being provided by the Medicines Healthcare Regulatory Authority (MHRA) for Pegcetacoplan (Aspaveli), NICE published its Final Guidance on Pegcetacoplan for treating PNH on 9 March 2022. Pegcetacoplan (Aspaveli) is now available to treat PNH patients on the NHS and is a possible treatment for paroxysmal nocturnal haemoglobinuria (PNH) in adults who have anaemia after at least 3 months of treatment with a C5 inhibitor.

The papers considered by the NICE committee for this appraisal are available here. These include PNH Support’s submission as part of this appraisal.

PNH Support welcomes innovate alternative treatments to improve the quality of life of PNH patients especially those who live with anaemia even when treated with C5 inhibitor treatments.