drugset
Drugs / Givosiran
last change Nov 2021 re-read 2 minutes ago

Givosiran

also known as ALN-AS1

Small interfering RNA (siRNA) targets ALAS1 via inhibition

Developed for
acute intermittent porphyria · hepatic porphyria · hereditary coproporphyria · porphyria due to ALA dehydratase deficiency · variegate porphyria
Investigated by
Alnylam Pharmaceuticals

Regulatory milestones approvals, filings & regulatory actions · 2 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Approved EU (EMA) Givlaari — 2020-03-02 europa.eu ↗
Approved US (FDA) GIVLAARI — 2019-11-20 fda.gov ↗

Trials 4 · a red edge is where a trial was stopped

today
US approved 2019
EU approved 2020
NCT03505853 · Ph 1 NCT03338816 · Ph 3 NCT02949830 · Ph 1/2
2017201820192020202120222023202420252026
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 11 trial
Phase 1 NCT03505853 Apr → Nov 2018 acute intermittent porphyria Alnylam Pharmaceuticals Completed No outcome recorded
Phase 1/21 trial
Phase 1/2 NCT02949830 Oct 2016 → Nov 2021 acute intermittent porphyria Alnylam Pharmaceuticals Completed No outcome recorded
Phase 31 trial
Phase 3 NCT03338816 Nov 2017 → Jan 2019 acute intermittent porphyria, hereditary coproporphyria, porphyria due to ALA dehydratase deficiency, variegate porphyria Alnylam Pharmaceuticals Completed No outcome recorded
Phase not stated1 trial
— NCT04056481 — hepatic porphyria Alnylam Pharmaceuticals Approved for marketing No outcome recorded
Also used as a comparator or background therapy in 1 trial
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1 NCT02452372 Comparator May 2015 → Sep 2017 acute intermittent porphyria Alnylam Pharmaceuticals Completed No outcome recorded

Press releases naming this drug 34 releases

DateIssuerRelease
2020-06-30 Alnylam Pharmaceuticals Alnylam Reports New 12-Month Interim Data From the ENVISION Phase 3 Study of Givosiran in Acute Hepatic Porphyria alnylam.com ↗
2020-06-10 Alnylam Pharmaceuticals Alnylam Announces Publication of ENVISION Phase 3 Study Results for Givosiran in the New England Journal of Medicine alnylam.com ↗
2020-03-03 Alnylam Pharmaceuticals Alnylam Announces Approval of GIVLAARI® (givosiran) in the European Union for the Treatment of Acute Hepatic Porphyria (AHP) in Adults and Adolescents alnylam.com ↗
2020-01-31 Alnylam Pharmaceuticals Alnylam Receives Positive CHMP Opinion for GIVLAARI™ (givosiran) for the Treatment of Acute Hepatic Porphyria in Adults and Adolescents alnylam.com ↗
2019-11-20 Alnylam Pharmaceuticals Alnylam Announces New and Enhanced Framework for Value-Based Agreements to Accelerate Patient and Provider Access to GIVLAARI™ (givosiran) alnylam.com ↗
2019-11-20 Alnylam Pharmaceuticals Alnylam Announces Approval of GIVLAARI™ (givosiran) by the U.S. Food and Drug Administration (FDA) alnylam.com ↗
2019-09-10 Alnylam Pharmaceuticals Alnylam Presents New Clinical Results for Givosiran at the 2019 International Congress on Porphyrins and Porphyrias alnylam.com ↗
2019-08-14 Alnylam Pharmaceuticals Alnylam to Report New Clinical Results for Givosiran at the 2019 International Congress on Porphyrins and Porphyrias alnylam.com ↗
2019-08-13 Alnylam Pharmaceuticals Alnylam Pharmaceuticals and Ironwood Pharmaceuticals Enter U.S. GI Disease Education and Promotional Agreement for Alnylam’s Givosiran in Acute Hepatic Porphyria (AHP) alnylam.com ↗
2019-08-05 Alnylam Pharmaceuticals Alnylam Announces U.S. Food and Drug Administration (FDA) Granted Priority Review of the Givosiran New Drug Application (NDA) for the Treatment of Acute Hepatic Porphyria alnylam.com ↗
2019-07-01 Alnylam Pharmaceuticals Alnylam Submits Marketing Authorization Application to the European Medicines Agency for Givosiran for the Treatment of Acute Hepatic Porphyria alnylam.com ↗
2019-06-05 Alnylam Pharmaceuticals Alnylam Completes Rolling Submission of New Drug Application to U.S. Food and Drug Administration (FDA) for Givosiran for the Treatment of Acute Hepatic Porphyria alnylam.com ↗

Evidence & citations 6 cited values

Every value below carries the sentence it was read from. 6 sources stand behind the page.

FieldValueCited text
Known as Givosiran ClinicalTrials.gov intervention name — accepted as the source's own label NCT03505853 ↗
4

NCT04056481 ↗

NCT02949830 ↗

NCT03338816 ↗

NCT02452372 ↗
Known as ALN-AS1 “givosiran (ALN-AS1) administered as a subcutaneous (SC) injection” NCT04056481 ↗
1

“givosiran (ALN-AS1)” NCT02452372 ↗
Action Inhibit “inhibits hepatic ALAS1 synthesis” PMID 30726693 ↗ Feb 2019
Modality Small interfering RNA (siRNA) “RNA interference therapeutic agent” PMID 30726693 ↗ Feb 2019
Route Subcutaneous “Single or multiple doses of ALN-AS1 by subcutaneous (sc) injection” NCT02452372 ↗
Target ALAS1 “inhibits hepatic ALAS1 synthesis” PMID 30726693 ↗ Feb 2019