NCT03338816 - Givosiran & Acute Intermittent Porphyria

Up-regulation of ALAS1 is key in acute hepatic porphyria. It causes ALA and porphobilinogen to build up. This leads to acute attacks and chronic symptoms. Givosiran is an RNA therapy. It blocks ALAS1 expression.
Balwani et al. 2020 NEJM 382: 2289-2301

This was a double-blind, placebo-controlled trial evaluating givosiran or placebo in patients with acute hepatic porphyria.
Balwani et al. 2020 NEJM 382: 2289-2301

Primary outcome measures:
The main goal was to measure porphyria attacks yearly. This was for patients with acute intermittent porphyria. Attacks led to hospitalization or urgent care. They also required hemin at home.
Balwani et al. 2020 NEJM 382: 2289-2301

Conclusion:
Givosiran reduced porph…