NICE backs Ultomiris for adult patients with PNH

15 April 2021

US biotech firm Alexion Pharmaceuticals (Nasdaq: ALXN) today announced the UK’s National Institute for Care and Health Excellence’s (NICE) decision to recommend Ultomiris (ravulizumab) for use in the National Health Service (NHS) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adult patients with hemolysis with clinical symptoms suggesting high disease activity, or whose disease is clinically stable after having Soliris (eculizumab) for at least six months.

Ravulizumab is the first approved, long-acting complement inhibitor that provides immediate, complete, and sustained C5 inhibition with only six or seven infusions per year instead of 26 with eculizumab, while delivering comparable safety and efficacy, said Alexion, which is currently the subject of a $39 billion takeover bid from UK pharma major AstraZeneca (LSE: AZN) that was announced in December.

The drug’s sales are expected to reach $1.66 billion by 2022, according to IBES data from Refinitiv. Ultomiris is expected to become the successor in PNH to Alexion's own Soliris.

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