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Page 1
Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria.
Balwani M, Sardh E, Ventura P, Peiró PA, Rees DC, Stölzel U, Bissell DM, Bonkovsky HL, Windyga J, Anderson KE, Parker C, Silver SM, Keel SB, Wang JD, Stein PE, Harper P, Vassiliou D, Wang B, Phillips J, Ivanova A, Langendonk JG, Kauppinen R, Minder E, Horie Y, Penz C, Chen J, Liu S, Ko JJ, Sweetser MT, Garg P, Vaishnaw A, Kim JB, Simon AR, Gouya L; ENVISION Investigators. Balwani M, et al. N Engl J Med. 2020 Jun 11;382(24):2289-2301. doi: 10.1056/NEJMoa1913147. N Engl J Med. 2020. PMID: 32521132 Clinical Trial.
RESULTS: A total of 94 patients underwent randomization (48 in the givosiran group and 46 in the placebo group). Among the 89 patients with acute intermittent porphyria, the mean annualized attack rate was 3.2 in the givosiran group and 12.5 in the placebo group, re …
RESULTS: A total of 94 patients underwent randomization (48 in the givosiran group and 46 in the placebo group). Among the 89 patient …
Efficacy and safety of givosiran for acute hepatic porphyria: 24-month interim analysis of the randomized phase 3 ENVISION study.
Ventura P, Bonkovsky HL, Gouya L, Aguilera-Peiró P, Montgomery Bissell D, Stein PE, Balwani M, Anderson DKE, Parker C, Kuter DJ, Monroy S, Oh J, Ritchie B, Ko JJ, Hua Z, Sweetser MT, Sardh E; ENVISION Investigators. Ventura P, et al. Liver Int. 2022 Jan;42(1):161-172. doi: 10.1111/liv.15090. Epub 2021 Nov 16. Liver Int. 2022. PMID: 34717041 Free PMC article. Clinical Trial.
Patients with acute hepatic porphyria (12 years) with recurrent attacks received givosiran (2.5 mg/kg monthly) (n = 48) or placebo (n = 46) for 6 months (double-blind period); 93 received givosiran (2.5 mg or 1.25 mg/kg monthly) in the open-label extension (continuo …
Patients with acute hepatic porphyria (12 years) with recurrent attacks received givosiran (2.5 mg/kg monthly) (n = 48) or placebo (n …
Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study.
Wang B, Ventura P, Takase KI, Thapar M, Cassiman D, Kubisch I, Liu S, Sweetser MT, Balwani M. Wang B, et al. Orphanet J Rare Dis. 2022 Aug 26;17(1):327. doi: 10.1186/s13023-022-02463-x. Orphanet J Rare Dis. 2022. PMID: 36028858 Free PMC article. Clinical Trial.
RESULTS: Participants (placebo, n = 46; givosiran, n = 48) included patients with low and high annualized attack rates (AARs; range 0-46). ...CONCLUSIONS: Patients with AHP, regardless of annualized attack rates, have considerable disease burden that may partly be alleviat …
RESULTS: Participants (placebo, n = 46; givosiran, n = 48) included patients with low and high annualized attack rates (AARs; range 0 …
Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria.
Sardh E, Harper P, Balwani M, Stein P, Rees D, Bissell DM, Desnick R, Parker C, Phillips J, Bonkovsky HL, Vassiliou D, Penz C, Chan-Daniels A, He Q, Querbes W, Fitzgerald K, Kim JB, Garg P, Vaishnaw A, Simon AR, Anderson KE. Sardh E, et al. N Engl J Med. 2019 Feb 7;380(6):549-558. doi: 10.1056/NEJMoa1807838. N Engl J Med. 2019. PMID: 30726693 Clinical Trial.
METHODS: We conducted a phase 1 trial of givosiran in patients with acute intermittent porphyria. 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 in …
METHODS: We conducted a phase 1 trial of givosiran in patients with acute intermittent porphyria. In part A of the trial, patients wi …
Pharmacokinetics and Pharmacodynamics of the Small Interfering Ribonucleic Acid, Givosiran, in Patients With Acute Hepatic Porphyria.
Agarwal S, Simon AR, Goel V, Habtemariam BA, Clausen VA, Kim JB, Robbie GJ. Agarwal S, et al. Clin Pharmacol Ther. 2020 Jul;108(1):63-72. doi: 10.1002/cpt.1802. Epub 2020 Mar 7. Clin Pharmacol Ther. 2020. PMID: 31994716 Clinical Trial.
Givosiran is a small interfering ribonucleic acid agent that was recently approved in the United States for the treatment of acute hepatic porphyria (AHP). ...Plasma exposures of AS(N-1)3' givosiran, an active metabolite with equal potency as givosiran, was 3 …
Givosiran is a small interfering ribonucleic acid agent that was recently approved in the United States for the treatment of acute he …
A Drug-Drug Interaction Study Evaluating the Effect of Givosiran, a Small Interfering Ribonucleic Acid, on Cytochrome P450 Activity in the Liver.
Vassiliou D, Sardh E, Harper P, Simon AR, Clausen VA, Najafian N, Robbie GJ, Agarwal S. Vassiliou D, et al. Clin Pharmacol Ther. 2021 Nov;110(5):1250-1260. doi: 10.1002/cpt.2419. Epub 2021 Sep 28. Clin Pharmacol Ther. 2021. PMID: 34510420 Clinical Trial.
The pharmacodynamic effect of givosiran (lowering of hepatic ALAS1, the first and rate limiting enzyme in the heme biosynthesis pathway) presents a unique scenario where givosiran could potentially impact heme-dependent activities in the liver, such as CYP enzyme ac …
The pharmacodynamic effect of givosiran (lowering of hepatic ALAS1, the first and rate limiting enzyme in the heme biosynthesis pathw …
A randomized, placebo-controlled study of givosiran in patients with acute hepatic porphyrias (ENVISION): Final (36-month) analysis of the Taiwan Cohort.
Lee MJ, Kuo HC, Chou LN, Sweetser MT, Wang JD. Lee MJ, et al. J Formos Med Assoc. 2024 Jun;123(6):679-686. doi: 10.1016/j.jfma.2023.10.016. Epub 2023 Dec 2. J Formos Med Assoc. 2024. PMID: 38044204 Free article. Clinical Trial.
This analysis was conducted to examine the long-term efficacy and safety of givosiran in Taiwanese participants in the ENVISION study (NCT03338816). ...Patients then switched from placebo to givosiran (placebo crossover group) or continued taking givosiran (c …
This analysis was conducted to examine the long-term efficacy and safety of givosiran in Taiwanese participants in the ENVISION study …