10 Sep, 2019 New Clinical Results for Givosiran at ICPP
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Capella
by Chris Brickley
We presented additional results from the ENVISION Phase 3 study and the Phase 1/2 open-label extension (OLE) study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of acute hepatic porphyria (AHP), at the 2019 International Congress on Porphyrins and Porphyrias (ICPP), held September 8-11 in Milan, Italy.
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Gouya et al. – “ENVISION, a Phase 3 Study to Evaluate the Efficacy and Safety of Givosiran, an Investigational RNAi Therapeutic Targeting Aminolevulinic Acid Synthase 1, in Acute Hepatic Porphyria Patients”
Sardh et al. – “Overall Health, Daily Functioning, and Quality of Life in Patients with Acute Hepatic Porphyria: ENVISION, a Phase 3 Global, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial”
Balwani et al. – “Disease Characteristics of Patients with Acute Hepatic Porphyria Patients: ENVISION, a Phase 3 Global, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial”
Bonkovsky et al. – “A Phase 1/2 Open-Label Extension Study of Givosiran, an Investigational RNAi Therapeutic, in Patients with Acute Intermittent Porphyria”
Anderson et al. – “Acute Hepatic Porphyria (AHP) Disease Manifestations and Daily Life Impacts in EXPLORE International Prospective, Natural History Study”
Gill et al. – “Burden of Illness in Acute Hepatic Porphyria (AHP): Insights from Patient and Caregiver Members of the British Porphyria Association”
Gill et al. – “The Evolving Diagnosis and Care of Patients with Acute Hepatic Porphyria (AHP) in the UK: from 2006 to 2018”
Vassiliou et al. – “A Drug-Drug Interaction Study to Investigate the Effect of Givosiran on the Activity of 5 Major Drug Metabolizing CYP450 Enzymes in Subjects with Acute Intermittent Porphyria (AIP) who are Chronic High Excreters (CHE)”
Porphyria attack reductions observed in the ENVISION Phase 3 study were maintained with ongoing givosiran dosing in the OLE phase of the study. Givosiran’s safety profile in the OLE phase has remained consistent with the profile observed in the double-blind phase of the ENVISION study.
In the ENVISION Phase 3 study, patients receiving givosiran reported reduced daily worst pain, a cardinal porphyria symptom, and givosiran was associated with improvements in patient-reported quality of life measures, compared to placebo.
In the Phase 1/2 OLE study, givosiran treatment of up to 30 months demonstrated sustained clinical activity with >90% decrease in mean annualized porphyria attack rate relative to baseline.
