Drugs / Lumasiran
Lumasiran
also known as
AD-65585 · AD-65585 SODIUM · ALN-65585 · ALN-65585 SODIUM · ALN-G01 · ALN-G01 SODIUM · ALN-GO1 · ALN-GO1 SODIUM · +2 more
Regulatory milestones approvals, filings & regulatory actions · 9 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed primary hyperoxaluria type 1 6 milestones | |||||
| Filed | US (FDA) | — | primary hyperoxaluria type 1 | 2020-10-22 | “Alnylam has filed a New Drug Application (NDA) for lumasiran with the FDA” alnylam.com ↗ |
| Filed | EU (EMA) | — | primary hyperoxaluria type 1 | 2020-05-26 | “the Marketing Authorisation Application (MAA) for lumasiran has been submitted to and validated by the European Medicines Agency (EMA)” alnylam.com ↗ |
| Filed | US (FDA) | — | primary hyperoxaluria type 1 | 2020-05-26 | “the U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug Application (NDA) for lumasiran” alnylam.com ↗ |
| Filed | EU (EMA) | — | primary hyperoxaluria type 1 | 2020-04-07 | “The Company also announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for lumasiran for the treatment of PH1.” alnylam.com ↗ |
| Filed | US (FDA) | — | primary hyperoxaluria type 1 | 2020-04-07 | “completion of the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for lumasiran” alnylam.com ↗ |
| Filed | US (FDA) | — | primary hyperoxaluria type 1 | 2020-01-10 | “initiated a rolling submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration ( FDA ) for lumasiran” alnylam.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansion | US (FDA) | OXLUMO | — | 2022-10-06 | fda.gov ↗ |
| Approved | US (FDA) | OXLUMO | — | 2020-11-23 | fda.gov ↗ |
| Approved | EU (EMA) | Oxlumo | — | 2020-11-19 | europa.eu ↗ |
Trials 8
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial · 1 met primary | ||||||
| Phase 1/2 | NCT02706886 | Mar 2016 → Jan 2019 | primary hyperoxaluria type 1 | Alnylam Pharmaceuticals | Completed | Met primary |
| Phase 23 trials | ||||||
| Phase 2 | NCT06225544 | Apr 2024 → Jan 2025 overdue | cardiovascular disorder, end stage renal failure | Charite University, Berlin, Germany | Recruiting | No outcome recorded |
| Phase 2 | NCT05161936 | Jan → Nov 2022 | nephrolithiasis, calcium oxalate | Alnylam Pharmaceuticals | Terminated | No outcome recorded Stop: Enrollment |
| Phase 2 | NCT03350451 | Apr 2018 → Feb 2023 | primary hyperoxaluria | Alnylam Pharmaceuticals | Completed | No outcome recorded |
| Phase 33 trials · 2 met primary | ||||||
| Phase 3 | NCT04152200 | Jan 2020 → May 2021 | primary hyperoxaluria type 1 | Alnylam Pharmaceuticals | Completed | Met primary |
| Phase 3 | NCT03905694 | Apr 2019 → Jun 2020 | primary hyperoxaluria type 1 | Alnylam Pharmaceuticals | Completed | No outcome recorded |
| Phase 3 | NCT03681184 | Nov 2018 → Nov 2019 | primary hyperoxaluria type 1 | Alnylam Pharmaceuticals | Completed | Met primary |
| Phase not stated1 trial | ||||||
| — | NCT04125472 | — | primary hyperoxaluria type 1 | Alnylam Pharmaceuticals | Approved for marketing | No outcome recorded |
News releases announcing trial results or a regulatory action · 27
| Date | Issuer | Release |
|---|---|---|
| 2022-10-06 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces FDA Approval of Supplemental New Drug Application for OXLUMO® (lumasiran) in Advanced Primary Hyperoxaluria Type 1 alnylam.com ↗ |
| 2022-05-24 | Alnylam Pharmaceuticals | Results Alnylam Presents New Results from the ILLUMINATE-C Phase 3 Study of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 alnylam.com ↗
Results from new exploratory analyses provide evidence that lumasiran treatment resulted in improvements in cardiac measures, nephrocalcinosis and kidney stone events. |
| 2022-03-01 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces U.S. Food and Drug Administration Acceptance of Supplemental New Drug Application for OXLUMO® for the Treatment of Advanced Primary Hyperoxaluria Type 1 alnylam.com ↗
The FDA has set an action date of October 6, 2022 , under the Prescription Drug User Fee Act (PDUFA). |
| 2021-12-14 | Alnylam Pharmaceuticals | Regulatory Alnylam Submits Regulatory Applications to the U.S. Food and Drug Administration and European Medicines Agency to Support Label Expansion for OXLUMO® for the Treatment of Advanced Primary Hyperoxaluria Type 1 alnylam.com ↗
The applications to the FDA and EMA are based on positive results from the ILLUMINATE-C single-arm, open-label Phase 3 study of lumasiran in patients of all ages with advanced PH1. |
| 2021-11-05 | Alnylam Pharmaceuticals | Results Alnylam Presents Positive Results from ILLUMINATE-C Phase 3 Study of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 alnylam.com ↗ |
| 2021-07-29 | Alnylam Pharmaceuticals | Results Alnylam Reports Positive Topline Results from ILLUMINATE-C Phase 3 Study of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today positive topline results from the ILLUMINATE-C Phase 3 open-label study of lumasiran in patients of all ages with advanced primary hyperoxaluria type 1 (PH1) associated with progressive decline in renal function. |
| 2021-05-03 | Alnylam Pharmaceuticals | Results Alnylam Announces Positive Early Results on Clinical Outcome Measures from ILLUMINATE-A Phase 3 Study of OXLUMO® (lumasiran) alnylam.com ↗
New results from ILLUMINATE-A showed that treatment with OXLUMO for 12 months was associated with evidence of improvements in nephrocalcinosis in one or both kidneys, relative to baseline. |
| 2021-03-31 | Alnylam Pharmaceuticals | Results Alnylam Announces Publication of ILLUMINATE-A Phase 3 Study Results for Lumasiran in The New England Journal of Medicine alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that pivotal trial results from the ILLUMINATE-A Phase 3 study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – for the treatment of primary hyperoxaluria type 1 (PH1), were published online in The New England Journal of Medicine ( NEJM ). |
| 2020-11-24 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces U.S. Food and Drug Administration (FDA) Approval of OXLUMO™ (lumasiran), the First and Only Treatment Approved for Primary Hyperoxaluria Type 1 to Lower Urinary Oxalate Levels in Pediatric and Adult Patients alnylam.com ↗
the U.S. Food and Drug Administration (FDA) approved OXLUMO™ (lumasiran) injection for subcutaneous use |
| 2020-11-19 | Alnylam Pharmaceuticals | Regulatory Alnylam Receives Approval for OXLUMO™ (lumasiran) in the European Union for the Treatment of Primary Hyperoxaluria Type 1 in All Age Groups alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the European Commission (EC) has granted marketing authorization for OXLUMO™ (lumasiran), an RNAi therapeutic, for the treatment of primary hyperoxaluria type 1 (PH1) in all age groups. |
| 2020-10-22 | Alnylam Pharmaceuticals | Results Alnylam Presents Positive Results from ILLUMINATE-B Phase 3 Study in Pediatric Patients with Primary Hyperoxaluria Type 1 at the American Society of Nephrology Kidney Week alnylam.com ↗ |
| 2020-10-16 | Alnylam Pharmaceuticals | Regulatory Alnylam Receives Positive CHMP Opinion for OXLUMO™ (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1 in All Age Groups alnylam.com ↗ |
| 2020-09-30 | Alnylam Pharmaceuticals | Results Alnylam Reports Positive Topline Results from ILLUMINATE-B Phase 3 Study of Lumasiran for the Treatment of Primary Hyperoxaluria Type 1 in Children Under the Age of Six alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today positive topline results from the ILLUMINATE-B pediatric Phase 3 study of lumasiran, an investigational RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – in development for the treatment of primary hyperoxaluria type 1 (PH1). |
| 2020-08-06 | Alnylam Pharmaceuticals | Results Alnylam Pharmaceuticals Reports Second Quarter 2020 Financial Results and Highlights Recent Period Activity alnylam.com ↗ |
| 2020-07-13 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces that the United Kingdom’s MHRA Grants Early Access to Lumasiran alnylam.com ↗
Positive Scientific Opinion Under Early Access to Medicines Scheme (EAMS) Will Make Lumasiran Treatment Available for UK Primary Hyperoxaluria Type 1 (PH1) Patients Before Marketing Authorization |
| 2020-06-07 | Alnylam Pharmaceuticals | Results Alnylam Presents Positive Phase 3 Results from ILLUMINATE-A Study of Lumasiran, an Investigational RNAi Therapeutic for Treatment of Primary Hyperoxaluria Type 1 alnylam.com ↗
Lumasiran achieved the ILLUMINATE-A primary endpoint with a 53.5 percent mean reduction in urinary oxalate relative to placebo (p=1.7x10 -14 ) and showed a 65.4 percent mean reduction in urinary oxalate relative to baseline. |
| 2020-05-26 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces U.S. Food and Drug Administration Has Granted Priority Review of the Lumasiran New Drug Application for the Treatment of Primary Hyperoxaluria Type 1 alnylam.com ↗ |
| 2020-05-06 | Alnylam Pharmaceuticals | Regulatory Alnylam Pharmaceuticals Reports First Quarter 2020 Financial Results and Highlights Recent Period Activity alnylam.com ↗
Completed New Drug Application and Filed Marketing Authorisation Application for Lumasiran, Advancing Another Alnylam Investigational RNAi Therapeutic toward Market |
| 2020-04-07 | Alnylam Pharmaceuticals | Regulatory Alnylam Completes Rolling Submission of New Drug Application to the U.S. Food and Drug Administration and Submits Marketing Authorization Application to the European Medicines Agency for Lumasiran for the Treatment of Primary Hyperoxaluria Type 1 alnylam.com ↗
The Company also announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for lumasiran for the treatment of PH1. |
| 2020-01-10 | Alnylam Pharmaceuticals | Regulatory Alnylam Initiates Rolling Submission of New Drug Application (NDA) to U.S. Food and Drug Administration (FDA) for Lumasiran for the Treatment of Primary Hyperoxaluria Type 1 (PH1) alnylam.com ↗
Specifically, Alnylam has submitted the non-clinical components to the FDA and expects to submit the remaining components in early 2020. |
| 2019-12-17 | Alnylam Pharmaceuticals | Results Alnylam Reports Positive Topline Results from ILLUMINATE-A Phase 3 Study of Lumasiran for the Treatment of Primary Hyperoxaluria Type 1 alnylam.com ↗
Specifically, lumasiran met the primary efficacy endpoint of percent change from baseline, relative to placebo, in 24-hour urinary oxalate excretion averaged across months 3 to 6 (p less than 0.0001). |
| 2019-11-09 | Alnylam Pharmaceuticals | Results Alnylam Initiates ILLUMINATE-C Phase 3 Study of Lumasiran for the Treatment of Advanced Primary Hyperoxaluria Type 1 and Presents New Positive Results from Phase 2 Open-Label Extension Study alnylam.com ↗
The Company also announced new positive efficacy results from the ongoing Phase 2 open-label extension (OLE) study of lumasiran, which were presented at the American Society of Nephrology (ASN) 2019 Annual Meeting on Saturday, November 9 in Washington, DC . |
| 2018-10-25 | Alnylam Pharmaceuticals | Results Alnylam Expands Alnylam Act® Program to Improve Diagnosis of Primary Hyperoxaluria Type 1 (PH1) and Aligns with FDA on Trial Design for ILLUMINATE-B Phase 3 Pediatric Study of Lumasiran alnylam.com ↗
Company Reports on New Phase 1/2 Results That Demonstrate Sustained Reductions in Both Urinary and Plasma Oxalate |
| 2018-10-04 | Alnylam Pharmaceuticals | Results Alnylam Reports Updated Positive Results from Phase 1/2 Study of Lumasiran in Patients with Primary Hyperoxaluria Type 1 alnylam.com ↗
Lumasiran demonstrated a mean maximal reduction in urinary oxalate of 75 percent (range: 43-87 percent) relative to baseline across cohorts dosed at 1 mg/kg monthly or 3 mg/kg monthly or quarterly (N=20). |
| 2018-06-08 | Alnylam Pharmaceuticals | Results Alnylam Reports Updated Positive Results from Phase 1/2 Study of Lumasiran in Patients with Primary Hyperoxaluria Type 1 (PH1) alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today new positive results from its Phase 1/2 study with lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of Primary Hyperoxaluria Type 1 (PH1). |
| 2018-03-26 | Alnylam Pharmaceuticals | Regulatory Alnylam Receives European Medicines Agency PRIME Designation for Accelerated Assessment of Lumasiran, an Investigational RNAi Therapeutic for the Treatment of Primary Hyperoxaluria Type 1 (PH1) 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 lumasiran (ALN-GO1), an investigational RNAi therapeutic targeting glycolate oxidase for the treatment of Primary Hyperoxaluria Type 1 (PH1). |
| 2017-11-03 | Alnylam Pharmaceuticals | Results Alnylam Reports Positive Preliminary Results from Ongoing Phase 1/2 Study of Lumasiran (ALN-GO1) in Patients with Primary Hyperoxaluria Type 1 (PH1) alnylam.com ↗ |
All press releases naming this drug 39 releases
Evidence & citations 14 cited values
Every value below carries the sentence it was read from. 10 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Lumasiran | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02706886 ↗ |
| Known as | AD-65585 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297779 ↗ |
| Known as | AD-65585 SODIUM | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297779 ↗ |
| Known as | ALN-65585 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297779 ↗ |
| Known as | ALN-65585 SODIUM | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297779 ↗ |
| Known as | ALN-G01 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297779 ↗ |
| Known as | ALN-G01 SODIUM | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297779 ↗ |
| Known as | ALN-GO1 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297779 ↗ |
| Known as | ALN-GO1 SODIUM | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297779 ↗ |
| Known as | LUMASIRAN SODIUM | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297779 ↗ |
| Known as | Oxlumo | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297779 ↗ |
| Action | Inhibit | “Lumasiran, an RNA interference therapeutic, suppresses glycolate oxidase, reducing hepatic oxalate production.” PMID 33985991 ↗ May 2021 |
| Modality | Small interfering RNA (siRNA) | “Lumasiran, an RNA interference therapeutic, suppresses glycolate oxidase, reducing hepatic oxalate production.” PMID 33985991 ↗ May 2021 |
| Route | Subcutaneous | “Lumasiran will be administered SC once in Part A.” NCT02706886 ↗ |