Givosiran

Unassigned

New Medicines

Acute hepatic porphyrias (AHP)

Information

New molecular entity
Alnylam Pharmaceuticals
Alnylam Pharmaceuticals

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1)
AHPs constitute a subset where the enzyme deficiency occurs within the liver, and includes acute intermittent porphyria (AIP), hereditary coproporphyria (HCP), and variegate porphyria (VP). Exposure of AHP patients to certain drugs, dieting, or hormonal changes can trigger strong induction of aminolevulinic acid synthase 1 (ALAS1), another enzyme in the heme biosynthesis pathway, which can lead to accumulation of neurotoxic heme intermediates [1]. Prevalence 1/75,000 [2].
Acute hepatic porphyrias (AHP)
Peri-tumoural and peri-lymphatic

Evidence based evaluations

NIHR