A review of the treatment landscape in paroxysmal nocturnal haemoglobinuria: where are we now and where are we going?

ABSTRACT: Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra-orphan disease, which until 15 years ago had limited treatment options. Eculizumab, a monoclonal antibody that inhibits C5 in the terminal complement cascade, has revolutionised treatment for this disease, near normalising life expectancy and improving quality of life for patients. The treatment landscape of PNH is now evolving, with ravulizumab a second longer acting intravenous C5 inhibitor now licenced by the FDA and EMA. With different therapeutic targets in the complement cascade and difference modalities of treatment, including subcutaneous, oral and intravenous therapies being developed, increasing independence for patients and reducing healthcare requirements. This review discusses the current and future therapies for PNH.

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SUBMITTER: Griffin M 

PROVIDER: S-EPMC10032435 | biostudies-literature | 2020 Jan-Dec

REPOSITORIES: biostudies-literature