NICE recommends Alexion's long-acting Ultomiris for PNH

NICE has recommended Alexion’s long-acting Ultomiris (ravulizumab) for the rare disease paroxysmal nocturnal haemoglobinuria (PNH) in final draft guidance.

Ultomiris is Alexion’s follow-up to its rare diseases blockbuster Soliris (eculizumab), which allows for  an eight-week dosing schedule after a loading phase.

This is more patient-friendly than Soliris, which requires infusions every two weeks, a considerable burden on patients’ lives.

NICE noted in its guidance document that “patient experts explained that the fortnightly infusions make it difficult for people to work, socialise and join in with family life."

“They also highlighted the psychological effect of the fortnightly infusions because they are constantly reminded that they have an incurable disease," NICE added.

Arranging infusions can also be a logistical challenge and frequent visits from nurses can lead to scarring of the veins, NICE's patient experts said.

In the guidance that applies to England and Wales, NICE said the drug is recommended within its marketing authorisation for adults with haemolysis and whose disease is stable after six months’ treatment with Soliris.

The company has agreed to supply Ultomiris at a commercially confidential discount to its list price of £4,533 per vial of 300 mg/3ml concentration and £16,621 per 1,100mg/11ml concentrate.

Alexion, which is being acquired by AstraZeneca, has built a significant presence in rare diseases with Soliris but needs a follow-up as cheaper biosimilars are expected to hit the US market in 2025.

In Europe, biosimilar competition is expected to begin in 2022 after the European Patent Office rejected an attempt to extend its patent life at the end of 2019.

PNH is a debilitating ultra-rare blood disorder where the body’s complement system begins to destroy red blood cells (haemolysis).

Blood clots can occur throughout the body, which can result in organ damage and premature death.

It can affects around 16 people per million in the UK, affecting men and women of all races, backgrounds and ages without warning with an average age of onset in the early 30s.