Long-term follow-up of givosiran treatment in patients with acute intermittent porphyria from a phase 1/2, 48-month open-label extension study.

ABSTRACT:

Background

Acute hepatic porphyria is a group of multisystem disorders of which acute intermittent porphyria is the most common subtype. Givosiran, a subcutaneously administered RNA interference therapeutic targeting liver ALAS mRNA, is approved for treating these disorders. This Phase 1/2 open-label extension study (NCT02949830) evaluated the long-term safety and efficacy of givosiran in adults with acute intermittent porphyria, with follow-up of up to 48 months, which is the longest follow-up of givosiran treatment to date. Participants were adults aged 18-65 years who completed part C of the Phase 1 givosiran study (NCT2452372).

Methods

Enrollees received givosiran for up to 48 months. Primary and secondary endpoints included the incidence of adverse events, changes in ur

SUBMITTER: Sardh E 

PROVIDER: S-EPMC11448181 | biostudies-literature | 2024 Oct

REPOSITORIES: biostudies-literature