Givosiran for treating acute hepatic porphyria: Evaluation consultation document

In DRAFT guidance, the committee was minded not to recommend givosiran for treating acute hepatic porphyria in people ≥12 years, and it recommended that NICE requests further information from the company, which should be made available for second evaluation committee meeting.

SPS commentary:

The committee noted:

• No evidence or analysis was provided comparing givosiran with prophylactic haem arginate.
• It is uncertain how effective givosiran is in the long term and how it will be used in clinical practice.
• It is not known if the starting age for treatment and the number of people stopping treatment at  menopause affect cost effectiveness. 

 

National Institute for Health and Care Excellence