Literature DB >> 32034693

Givosiran: First Approval.

Lesley J Scott1.   

Abstract

Givosiran (Givlaari™) is an aminolevulinate synthase 1 (ALAS1)-directed small interfering RNA (siRNA) covalently linked to a ligand to enable specific delivery of the siRNA to hepatocytes. This results in downregulation of ALAS1 mRNA and prevents accumulation of neurotoxic δ-aminolevulinic acid and porphobilinogen levels that are associated with acute porphyria attacks. Givosiran is being developed by Alnylam Pharmaceuticals for the treatment of acute hepatic porphyria (AHP). In November 2019, givosiran was approved in the USA for the treatment of adults with AHP based on the positive results from the multinational, phase III ENVISION trial. In the EU, givosiran received a positive opinion in January 2020 for the treatment of AHP in adults and adolescents aged 12 years and older. This article summarizes the milestones in the development of givosiran leading to this first approval for the treatment of adults with AHP.

Entities:  

Mesh：

Substances：

Year:  2020        PMID： 32034693     DOI： 10.1007/s40265-020-01269-0

Source DB:  PubMed          Journal:  Drugs        ISSN： 0012-6667            Impact factor:   9.546


  8 in total

1.  The incidence of inherited porphyrias in Europe.

Authors:  George Elder; Pauline Harper; Michael Badminton; Sverre Sandberg; Jean-Charles Deybach
Journal:  J Inherit Metab Dis       Date:  2012-11-01       Impact factor: 4.982

Review 2.  Current and innovative emerging therapies for porphyrias with hepatic involvement.

Authors:  Antonio Fontanellas; Matías A Ávila; Karl E Anderson; Jean-Charles Deybach
Journal:  J Hepatol       Date:  2019-05-16       Impact factor: 25.083

Review 3.  Porphyria.

Authors:  D Montgomery Bissell; Karl E Anderson; Herbert L Bonkovsky
Journal:  N Engl J Med       Date:  2017-08-31       Impact factor: 91.245

4.  Recurrent attacks of acute hepatic porphyria: major role of the chronic inflammatory response in the liver.

Authors:  C Schmitt; H Lenglet; A Yu; C Delaby; A Benecke; T Lefebvre; P Letteron; V Paradis; S Wahlin; S Sandberg; P Harper; E Sardh; A K Sandvik; J R Hov; A K Aarsand; L Chiche; C Bazille; J-Y Scoazec; J To-Figueras; M Carrascal; J Abian; A Mirmiran; Z Karim; J-C Deybach; H Puy; K Peoc'h; H Manceau; L Gouya
Journal:  J Intern Med       Date:  2018-03-26       Impact factor: 8.989

5.  Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria.

Authors:  Eliane Sardh; Pauline Harper; Manisha Balwani; Penelope Stein; David Rees; D Montgomery Bissell; Robert Desnick; Charles Parker; John Phillips; Herbert L Bonkovsky; Daphne Vassiliou; Craig Penz; Amy Chan-Daniels; Qiuling He; William Querbes; Kevin Fitzgerald; Jae B Kim; Pushkal Garg; Akshay Vaishnaw; Amy R Simon; Karl E Anderson
Journal:  N Engl J Med       Date:  2019-02-07       Impact factor: 91.245

Review 6.  Acute Hepatic Porphyrias: Review and Recent Progress.

Authors:  Bruce Wang; Sean Rudnick; Brent Cengia; Herbert L Bonkovsky
Journal:  Hepatol Commun       Date:  2018-12-20

7.  EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent Attacks.

Authors:  Laurent Gouya; Paolo Ventura; Manisha Balwani; D Montgomery Bissell; David C Rees; Ulrich Stölzel; John D Phillips; Raili Kauppinen; Janneke G Langendonk; Robert J Desnick; Jean-Charles Deybach; Herbert L Bonkovsky; Charles Parker; Hetanshi Naik; Michael Badminton; Penelope E Stein; Elisabeth Minder; Jerzy Windyga; Radan Bruha; Maria Domenica Cappellini; Eliane Sardh; Pauline Harper; Sverre Sandberg; Aasne K Aarsand; Janice Andersen; Félix Alegre; Aneta Ivanova; Neila Talbi; Amy Chan; William Querbes; John Ko; Craig Penz; Shangbin Liu; Tim Lin; Amy Simon; Karl E Anderson
Journal:  Hepatology       Date:  2019-11-07       Impact factor: 17.425

8.  Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification.

Authors:  Amy Chan; Abigail Liebow; Makiko Yasuda; Lin Gan; Tim Racie; Martin Maier; Satya Kuchimanchi; Don Foster; Stuart Milstein; Klaus Charisse; Alfica Sehgal; Muthiah Manoharan; Rachel Meyers; Kevin Fitzgerald; Amy Simon; Robert J Desnick; William Querbes
Journal:  Mol Ther Nucleic Acids       Date:  2015-11-03       Impact factor: 10.183
  8 in total
  73 in total

Review 1.  Delivery of Oligonucleotides to the Liver with GalNAc: From Research to Registered Therapeutic Drug.

Authors:  Alexandre J Debacker; Jon Voutila; Matthew Catley; David Blakey; Nagy Habib
Journal:  Mol Ther       Date:  2020-06-17       Impact factor: 11.454

2.  Nanosac, a Noncationic and Soft Polyphenol Nanocapsule, Enables Systemic Delivery of siRNA to Solid Tumors.

Authors:  Hyungjun Kim; Simseok A Yuk; Alexandra M Dieterly; Soonbum Kwon; Jinho Park; Fanfei Meng; Hytham H Gadalla; Maria Jose Cadena; L Tiffany Lyle; Yoon Yeo
Journal:  ACS Nano       Date:  2021-03-01       Impact factor: 15.881

3.  Development of siRNA Therapeutics for the Treatment of Liver Diseases.

Authors:  Anja Holm; Marianne Bengtson Løvendorf; Sakari Kauppinen
Journal:  Methods Mol Biol       Date:  2021

4.  siRNA Therapeutics in Ocular Diseases.

Authors:  Javier Moreno-Montañés; Anne-Marie Bleau; Tamara Martínez; Beatriz Vargas; María Victoria González; Ana Isabel Jiménez
Journal:  Methods Mol Biol       Date:  2021

5.  Synthesis of GalNAc-Oligonucleotide Conjugates Using GalNAc Phosphoramidite and Triple-GalNAc CPG Solid Support.

Authors:  Egor A Ulashchik; Yury V Martynenko-Makaev; Tatsiana P Akhlamionok; Denis M Melnik; Vadim V Shmanai; Timofei S Zatsepin
Journal:  Methods Mol Biol       Date:  2021

6.  Next generation miRNA inhibition using short anti-seed PNAs encapsulated in PLGA nanoparticles.

Authors:  Shipra Malik; Jihoon Lim; Frank J Slack; Demetrios T Braddock; Raman Bahal
Journal:  J Control Release       Date:  2020-08-21       Impact factor: 9.776

7.  RNA Drugs and RNA Targets for Small Molecules: Principles, Progress, and Challenges.

Authors:  Ai-Ming Yu; Young Hee Choi; Mei-Juan Tu
Journal:  Pharmacol Rev       Date:  2020-10       Impact factor: 25.468

Review 8.  Antisense Drugs Make Sense for Neurological Diseases.

Authors:  C Frank Bennett; Holly B Kordasiewicz; Don W Cleveland
Journal:  Annu Rev Pharmacol Toxicol       Date:  2020-10-09       Impact factor: 13.820

9.  Immunotherapy for breast cancer using EpCAM aptamer tumor-targeted gene knockdown.

Authors:  Ying Zhang; Xuemei Xie; Pourya Naderi Yeganeh; Dian-Jang Lee; David Valle-Garcia; Karla F Meza-Sosa; Caroline Junqueira; Jiayu Su; Hongbo R Luo; Winston Hide; Judy Lieberman
Journal:  Proc Natl Acad Sci U S A       Date:  2021-03-02       Impact factor: 11.205

Review 10.  The evolution of commercial drug delivery technologies.

Authors:  Ava M Vargason; Aaron C Anselmo; Samir Mitragotri
Journal:  Nat Biomed Eng       Date:  2021-04-01       Impact factor: 25.671
View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.