givosiran

01/01/2021 - "Case report of a complicated neurologically manifesting acute porphyria treated successfully with Givosiran."
01/01/2021 - "Givosiran is an effective treatment for prevention of acute porphyria attacks in AHP patients with frequent recurrent attacks."
01/01/2020 - "This phase I study evaluated the safety, pharmacokinetic, and pharmacodynamic profile of subcutaneously (SC) administered givosiran in patients with acute intermittent porphyria, the most common AHP type. "
01/01/2019 - "We conducted a phase 1 trial of givosiran in patients with acute intermittent porphyria. "
11/24/2023 - "Acute intermittent porphyria: Acute flare-up resolved with givosiran."

05/01/2021 - "Givosiran also improved several other outcomes, including hemin use and pain (the cardinal symptom of AHP). "
01/01/2022 - "Givosiran reduced the number and severity of attacks, days with worst pain scores above baseline, and opioid use versus placebo. "
01/01/2022 - "We analyzed baseline AHP severity via chronic symptoms between attacks, comorbidities, concomitant medications, hemin-associated complications, and quality of life (QOL) and evaluated givosiran (2.5 mg/kg monthly) in patients with and without prior hemin prophylaxis on number and severity of attacks and pain scores during and between attacks. "
01/01/2022 - "Long-term givosiran has an acceptable safety profile and significantly benefits acute hepatic porphyria patients with recurrent attacks by reducing attack frequency, hemin use, and severity of daily worst pain while improving quality of life."
01/01/2022 - "Long-term givosiran led to sustained lowering of delta-aminolevulinic acid and porphobilinogen and improvements in daily worst pain and quality of life. "

06/13/2023 - "siRNA-based treatment for hereditary transthyretin-mediated amyloidosis (hATTR) using Onpattro and acute hepatic porphyria (AHP) by Givlaari and three more FDA-approved siRNA drugs has set up a milestone and further improved the confidence for the development of gene therapeutics for a spectrum of diseases. "
11/01/2023 - "Patisiran (hereditary transthyretin-mediated amyloidosis) was the first licensed siRNA, followed by givosiran (acute hepatic porphyria), lumasiran (primary hyperoxaluria type 1) and inclisiran (familial hypercholesterolaemia), which all use N-acetylgalactosamine (GalNAc) linkage for effective liver-directed delivery. "

05/01/2021 - "In the phase 3 ENVISION trial, givosiran significantly reduced the annualized rate of composite porphyria attacks (i.e. "
01/01/2022 - "Case Report: Lack of Response to Givosiran in a Case of ALAD Porphyria."
11/01/2020 - "Givosiran - Running RNA Interference to Fight Porphyria Attacks."
11/01/2020 - "Among patients with acute intermittent porphyria, those who received givosiran had a significantly lower rate of porphyria attacks and better results for multiple other disease manifestations than those who received placebo. "
05/01/2022 - "The case series documents the impact of a new mRNA interference molecule givosiran as well as a plan for embryo selection which is not commonly used in porphyria. "

01/01/2023 - "The PK of givosiran and its active metabolite were not significantly affected by demographic or clinical parameters that would require adjustment from the approved body weight-based dose of givosiran 2.5 mg/kg once monthly."
01/01/2023 - "Body weight, East Asian ethnicity, and renal impairment were significant covariates in the model; however, none of the covariates evaluated resulted in clinically meaningful differences in plasma exposures of givosiran and AS(N-1)3' givosiran. "
11/01/2020 - "In this double-blind, placebo-controlled, phase 3 trial, we randomly assigned symptomatic patients with acute hepatic porphyria to receive either subcutaneous givosiran (2.5 mg per kilogram of body weight) or placebo monthly for 6 months. "
01/01/2019 - "In part A of the trial, patients without recent porphyria attacks (i.e., no attacks in the 6 months before baseline) were randomly assigned to receive a single subcutaneous injection of one of five ascending doses of givosiran (0.035, 0.10, 0.35, 1.0, or 2.5 mg per kilogram of body weight) or placebo. "