1 Recommendations

1.1 Givosiran is recommended as an option for treating acute hepatic porphyria (AHP) in adults and young people aged 12 and older, only if:

  • they have clinically confirmed severe recurrent attacks (4¬†attacks or more within 12¬†months) and

  • the company provides it according to the commercial arrangement.

1.2 This recommendation is not intended to affect treatment with givosiran that was started in the NHS before this guidance was published. Adults and young people having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop. This decision should be made jointly by the clinician, the young person and their parents or carers.

Why the committee made these recommendations

AHP is a rare, progressive and potentially life-threatening condition that can significantly affect the quality of life of people with the condition, and their families and carers. People can have acute attacks with extreme pain, nausea and fatigue, which sometimes lead to seizures and paralysis. They can also have chronic pain and fatigue. Standard treatment in the NHS is prophylactic haem arginate, which is offered to most people with recurrent severe attacks despite this use being outside its marketing authorisation.

There is no trial directly comparing givosiran with prophylactic haem arginate. However, results from clinical studies and clinical expert opinion suggest that givosiran reduces the number of acute attacks in people with AHP, as well as improving chronic symptoms and quality of life.

Some assumptions in the economic modelling are uncertain, particularly around the duration of treatment with givosiran and the effectiveness of prophylactic haem arginate. Despite this, givosiran is likely to provide important clinical benefit and improve quality of life for people with AHP. It also provides value for money within the context of a highly specialised service. Givosiran is therefore recommended for use in the NHS.

  • National Institute for Health and Care Excellence (NICE)