Drugs / Givosiran
Regulatory milestones approvals, filings & regulatory actions · 6 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed hepatic cutaneous porphyria 3 milestones | |||||
| Filed | EU (EMA) | — | hepatic cutaneous porphyria | 2019-07-01 | “the Marketing Authorisation Application (MAA) for givosiran has been submitted to and validated by the European Medicines Agency (EMA)” alnylam.com ↗ “today announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for givosiran” alnylam.com ↗ |
| Filed | US (FDA) | — | hepatic cutaneous porphyria | 2019-06-05 | “announced today the completion of the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration ( FDA ) for givosiran” alnylam.com ↗ “the U.S. Food and Drug Administration ( FDA ) has accepted the Company’s New Drug Application (NDA) for givosiran” alnylam.com ↗ |
| Filed | US (FDA) | — | hepatic cutaneous porphyria | 2018-12-06 | “Initiated rolling submission of a New Drug Application (NDA) with plans to pursue full approval based on complete results” alnylam.com ↗ “In addition, Alnylam announces initiation of a rolling submission of an NDA to the FDA for givosiran” alnylam.com ↗ “Rolling submission for a new drug application (NDA) has been recently initiated” alnylam.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Filed | Other | — | — | 2020-08-06 | “Submitted a Marketing Authorization Application (MAA) for givosiran in Switzerland and Israel” alnylam.com ↗ “Submitted a Marketing Authorization Application (MAA) for givosiran in Switzerland” alnylam.com ↗ |
| Approved | EU (EMA) | Givlaari | — | 2020-03-02 | europa.eu ↗ |
| Approved | US (FDA) | GIVLAARI | — | 2019-11-20 | fda.gov ↗ |
Trials 4
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| 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 · 1 met primary | ||||||
| Phase 3 | NCT03338816 | Nov 2017 → Jan 2019 | acute intermittent porphyria, hereditary coproporphyria, porphyria due to ALA dehydratase deficiency, variegate porphyria | Alnylam Pharmaceuticals | Completed | Met primary |
| Phase not stated1 trial | ||||||
| — | NCT04056481 | — | hepatic porphyria | Alnylam Pharmaceuticals | Approved for marketing | No outcome recorded |
| Comparator or background therapy1 trial | ||||||
| Phase 1 | NCT02452372 Comparator | May 2015 → Sep 2017 | acute intermittent porphyria | Alnylam Pharmaceuticals | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 24
| Date | Issuer | Release |
|---|---|---|
| 2020-06-30 | Alnylam Pharmaceuticals | Results 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 | Results 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 | Regulatory Alnylam Announces Approval of GIVLAARI® (givosiran) in the European Union for the Treatment of Acute Hepatic Porphyria (AHP) in Adults and Adolescents alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the European Commission (EC) has granted marketing authorization for GIVLAARI ® (givosiran), an injection for subcutaneous use targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older. |
| 2020-01-31 | Alnylam Pharmaceuticals | Regulatory 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 | Regulatory Alnylam Announces Approval of GIVLAARI™ (givosiran) by the U.S. Food and Drug Administration (FDA) alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration ( FDA ) approved GIVLAARI ™ (givosiran) injection for subcutaneous use for the treatment of adults with acute hepatic porphyria (AHP). |
| 2019-09-10 | Alnylam Pharmaceuticals | Results Alnylam Presents New Clinical Results for Givosiran at the 2019 International Congress on Porphyrins and Porphyrias alnylam.com ↗
Porphyria Attack Reductions Observed in ENVISION Phase 3 Study Maintained with Ongoing Givosiran Dosing in Open-Label Extension (OLE) Study |
| 2019-08-05 | Alnylam Pharmaceuticals | Regulatory 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 ↗
The FDA has set an action date of February 4, 2020 under the Prescription Drug User Fee Act (PDUFA), and the agency has indicated that they are not currently planning an advisory committee meeting as part of the NDA review. |
| 2019-07-01 | Alnylam Pharmaceuticals | Regulatory 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 | Regulatory 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 ↗
We’re very pleased to have completed our NDA filing for givosiran, just 90 days since reporting our positive topline ENVISION Phase 3 study results, consistent with our commitment to bring givosiran to patients as rapidly as possible. |
| 2019-04-12 | Alnylam Pharmaceuticals | Results Alnylam Presents Positive Complete Results from ENVISION Phase 3 Study of Givosiran, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyria alnylam.com ↗
The full ENVISION results demonstrated a 74 percent mean and 90 percent median reduction in the primary endpoint measure of annualized rate of composite attacks in patients on givosiran relative to placebo during the six-month double-blind period. |
| 2019-03-06 | Alnylam Pharmaceuticals | Results Alnylam Reports Positive Topline Results from ENVISION Phase 3 Study of Givosiran in Acute Hepatic Porphyria alnylam.com ↗
We believe the ENVISION results demonstrate a robust therapeutic benefit of givosiran treatment on the debilitating aspects of this disease. |
| 2019-02-06 | Alnylam Pharmaceuticals | Results Alnylam Announces Publication of Phase 1 Givosiran Data in The New England Journal of Medicine alnylam.com ↗
In the Phase 1 study, the proportion of patients reporting adverse events (AEs) was similar across treatment groups with no clear relationship with givosiran dose. |
| 2018-12-06 | Alnylam Pharmaceuticals | Regulatory Alnylam Provides R&D Updates and Announces 2019 Product and Pipeline Goals at R&D Day alnylam.com ↗
Initiates Rolling Submission of Givosiran NDA to U.S. Food and Drug Administration ( FDA ) |
| 2018-11-09 | Alnylam Pharmaceuticals | Results Alnylam Presents Updated Phase 1/2 Open-Label Extension (OLE) Results for Givosiran, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyria alnylam.com ↗
Givosiran Demonstrated Sustained Clinical Activity, with an Over 90 Percent Decrease in Mean Annualized Porphyria Attack Rate, Relative to Baseline |
| 2018-10-15 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces Plan to Initiate Rolling Submission of a New Drug Application and Pursue Full Approval for Givosiran alnylam.com ↗
The FDA has also agreed to a rolling submission of a New Drug Application (NDA), which will be initiated in 2018 with full clinical sections submitted in mid-2019, assuming positive study results. |
| 2018-09-27 | Alnylam Pharmaceuticals | Results Alnylam Announces Positive Topline Results from Interim Analysis of ENVISION Phase 3 Study of Givosiran in Patients with Acute Hepatic Porphyria alnylam.com ↗
Results of the interim analysis showed that givosiran treatment was associated with a statistically significant reduction in urinary ALA levels in acute intermittent porphyria (AIP) patients, relative to placebo (p less than 0.001). |
| 2018-04-14 | Alnylam Pharmaceuticals | Results Alnylam Presents New Positive Clinical Results for Givosiran, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyrias alnylam.com ↗
These results were presented at the European Association for the Study of the Liver (EASL) 53 rd Annual International Liver Congress™, being held April 11-15, 2018 in Paris, France . |
| 2017-06-26 | Alnylam Pharmaceuticals | Results Alnylam Reports New Positive Clinical Results for Givosiran (ALN-AS1), an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyrias alnylam.com ↗
Results provide evidence that givosiran has the potential to prevent porphyria attacks in patients with acute intermittent porphyria (AIP) suffering with recurrent attacks. |
| 2017-05-31 | Alnylam Pharmaceuticals | Regulatory FDA Grants Breakthrough Therapy Designation for Alnylam's Givosiran for the Prophylaxis of Attacks in Patients with Acute Hepatic Porphyria alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that it has received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for givosiran (ALN-AS1) |
| 2017-03-01 | Alnylam Pharmaceuticals | Regulatory Alnylam Receives European Medicines Agency PRIME Designation for Accelerated Assessment of Givosiran, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyrias alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company, announced today that the European Medicines Agency (EMA) has granted access to its Priority Medicines (PRIME) scheme for givosiran (ALN-AS1), an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias. |
| 2016-12-03 | Alnylam Pharmaceuticals | Results Alnylam Reports Positive Initial Clinical Activity Results for Givosiran (ALN-AS1), an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyrias alnylam.com ↗ |
| 2015-01-05 | Alnylam Pharmaceuticals | Regulatory Alnylam Files Clinical Trial Application to Initiate a Phase 1 Study for ALN-AS1, a Subcutaneously Administered, Investigational RNAi Therapeutic Targeting Aminolevulinic Acid Synthase-1 (ALAS-1) for the Treatment of Hepatic Porphyrias alnylam.com ↗
announced today that it has filed a Clinical Trial Application (CTA) with the Swedish Medical Products Agency (MPA) |
| 2014-05-21 | Alnylam Pharmaceuticals | Results Alnylam and Collaborators Publish Pre-Clinical Results with ALN-AS1, an RNAi Therapeutic Targeting Aminolevulinic Acid Synthase-1 (ALAS-1) for the Treatment of Hepatic Porphyrias, in the Proceedings of the National Academy of Sciences alnylam.com ↗
This new paper provides proof of concept for an RNAi therapeutic for the treatment of AIP. |
| 2013-10-08 | Alnylam Pharmaceuticals | Results Alnylam Advances Development Candidate for ALN-AS1, a Subcutaneously Administered RNAi Therapeutic Targeting Aminolevulinate Synthase 1 (ALAS-1) for the Treatment of Hepatic Porphyrias alnylam.com ↗
Based on these findings, including results in non-human primate studies, the company has selected its ALN-AS1 Development Candidate and expects to file an Investigational New Drug (IND) application for this RNAi therapeutic in 2014. |
All press releases naming this drug 35 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 6 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Givosiran | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03338816 ↗ |
| 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 |