Regulatory milestones approvals, filings & regulatory actions · 7 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed hypercholesterolemia due to cholesterol 7alpha-hydroxylase deficiency 1 milestone | |||||
| Filed | US (FDA) | Leqvio | hypercholesterolemia due to cholesterol 7alpha-hydroxylase deficiency | 2021-07-21 | “Inclisiran (KJX839) was filed in the US for primary hyperlipidemia and in the EU for both primary hypercholesterolemia and mixed dyslipidemia” novartis.com ↗ “Resubmission to the FDA for the inclisiran New Drug Application (NDA) to address the Complete Response Letter (CRL) was filed” novartis.com ↗ “Inclisiran was submitted in the US for primary hyperlipidemia and in the EU for mixed dyslipidemia” novartis.com ↗ “Inclisiran resubmission of NDA filed with FDA to address manufacturing related CRL” novartis.com ↗ |
| Filed hypercholesterolemia, autosomal dominant, type B 1 milestone | |||||
| Filed | US (FDA) | — | hypercholesterolemia, autosomal dominant, type B | 2021-10-26 | “Der erneute Zulassungsantrag (New Drug Application, NDA) wurde bei der FDA eingereicht mit einer Prüfungsfrist bis zum 1. Januar 2022” novartis.com ↗ “Inclisiran Resubmission to the FDA for the inclisiran NDA was filed with an action date of January 1, 2022” novartis.com ↗ “In December 2019, The Medicines Company submitted the New Drug Application (NDA) for inclisiran to the FDA” novartis.com ↗ |
| Filed hyperlipidemia, combined, 2 1 milestone | |||||
| Filed | EU (EMA) | — | hyperlipidemia, combined, 2 | — | “Inclisiran (KJX839) was filed in the US for primary hyperlipidemia and in the EU for both primary hypercholesterolemia and mixed dyslipidemia” novartis.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansions (2) | US (FDA) | LEQVIO | — | 2025-07-31 – 2026-02-12 | fda.gov ↗ |
| Approved | US (FDA) | LEQVIO | — | 2021-12-22 | fda.gov ↗ |
| Approved | EU (EMA) | Leqvio | — | 2020-12-09 | europa.eu ↗ |
Trials 48
News releases announcing trial results or a regulatory action · 19
| Date | Issuer | Release |
|---|---|---|
| 2025-11-04 | Novartis Pharmaceuticals | Results New Novartis data at ASN Kidney Week and AHA Scientific Sessions demonstrate momentum of broad CRM portfolio and pipeline novartis.com ↗
V-INCEPTION analyses highlight Leqvio adherence and goal attainment data when initiated early in post-acute coronary syndrome patients |
| 2025-08-30 | Novartis Pharmaceuticals | Results Novartis Leqvio® shows statistically significant and clinically meaningful early LDL-C goal achievement with less muscle pain novartis.com ↗
After 90 days of treatment with Leqvio on top of LLT, 85% of patients achieved their guideline-recommended LDL-C target compared to 31% of those receiving placebo on top of LLT (p<0.0001) 1 . |
| 2024-08-28 | Novartis Pharmaceuticals | Results Novartis twice-yearly* Leqvio® demonstrated clinically meaningful, statistically significant LDL-C lowering as a monotherapy in patients at low or moderate ASCVD risk novartis.com ↗
Novartis announced today positive topline results from twice-yearly* Leqvio ® (inclisiran) in the Phase III V-MONO study, which met its primary endpoints. |
| 2024-04-06 | Novartis Pharmaceuticals | Results New Novartis data show early addition of twice-yearly* Leqvio® (inclisiran) following maximally tolerated statin therapy significantly reduces LDL-C in ASCVD patients in real-world setting novartis.com ↗
V-INITIATE trial demonstrates that early initiation with Leqvio, prior to guideline-recommended ezetimibe, for ASCVD patients unable to achieve LDL-C goal on statin therapy alone led to significant LDL-C reduction vs. clinician-determined usual care (60% vs. 7% respectively) 1 |
| 2023-08-28 | Novartis Pharmaceuticals | Results Novartis presents new long-term Leqvio® (inclisiran) data demonstrating consistent efficacy and safety beyond six years novartis.com ↗ |
| 2022-11-07 | Novartis Pharmaceuticals | Results New long-term Leqvio® (inclisiran) data from Novartis show sustained efficacy and safety over four years novartis.com ↗
Results from ORION-3 open-label trial show twice-yearly* Leqvio ® (inclisiran), as a complement to statin therapy, provides effective and sustained reductions in low-density lipoprotein cholesterol (LDL-C) over four years of treatment 1 ,2 |
| 2021-12-22 | Novartis Pharmaceuticals | Regulatory FDA approves Novartis Leqvio® (inclisiran), first-in-class siRNA to lower cholesterol and keep it low with two doses a year novartis.com ↗ |
| 2021-12-22 | Alnylam Pharmaceuticals | Regulatory Alnylam Comments on FDA Approval of Leqvio®, the First siRNA (RNAi Therapeutic) Approved to Reduce LDL-C alnylam.com ↗
Leqvio ® Becomes Fourth RNAi Therapeutic Approved by U.S. FDA and First and Only for Lowering LDL-C in Atherosclerotic Cardiovascular Disease (ASCVD) Which Can Affect Up to 30 Million Americans |
| 2021-11-08 | Novartis Pharmaceuticals | Results Leqvio®* (inclisiran) reduced LDL-C in people who are overweight or obese novartis.com ↗
Novartis today announced results from a pooled post-hoc analysis of Phase III ORION-9, -10 and -11 trials, exploring the impact of body mass index (BMI) levels on the efficacy and safety of twice-yearly** Leqvio ® (inclisiran). |
| 2021-08-30 | Novartis Pharmaceuticals | Results Novartis Leqvio®* (inclisiran) analyses show effective and sustained LDL-C reduction in two sub-populations of patients with ASCVD novartis.com ↗
In the first analysis, patients with established CeVD treated with Leqvio achieved an average 55.2% reduction in LDL-C from baseline to Day 510 compared with placebo ( P <.0001) 1 . |
| 2020-12-18 | Novartis Pharmaceuticals | Regulatory Novartis receives complete response letter from U.S. FDA for inclisiran novartis.com ↗
The U.S. Food and Drug Administration (FDA) has issued a complete response letter (CRL) regarding the new drug application (NDA) for inclisiran, a potential treatment for hyperlipidemia in adults who have elevated low-density lipoprotein cholesterol (LDL-C) while being on a maximum tolerated dose of a statin therapy. |
| 2020-11-13 | Novartis Pharmaceuticals | Results New Novartis analyses for investigational inclisiran demonstrate consistently effective and sustained LDL-C reduction at month 17 regardless of age and gender novartis.com ↗ |
| 2020-11-05 | Alnylam Pharmaceuticals | Regulatory Alnylam Pharmaceuticals Reports Third Quarter 2020 Financial Results and Highlights Recent Period Activity alnylam.com ↗
Received a positive CHMP opinion from EMA recommending approval of inclisiran for the treatment of adults with hypercholesterolemia or mixed dyslipidemia. |
| 2020-10-16 | Novartis Pharmaceuticals | Regulatory Novartis receives positive CHMP opinion for Leqvio®* (inclisiran), a potential first-in-class siRNA for the treatment of high cholesterol novartis.com ↗ |
| 2020-03-28 | Novartis Pharmaceuticals | Results Novartis new analysis further shows durable and potent LDL-C reduction with inclisiran, an investigational first-in-class siRNA cholesterol-lowering treatment novartis.com ↗
The pooled analysis of the ORION-9, -10 and -11 Phase III trials showed a durable and potent reduction in LDL-C of 51% when used in addition to other lipid-lowering therapies (LLT) over 17 months of treatment 1 . |
| 2017-08-28 | Alnylam Pharmaceuticals | Results New, One-Year Data from ORION-1 Phase II Study of Inclisiran Extends Excellent Long-Term Efficacy and Safety Profile, Affirming Dose for Phase III Trials alnylam.com ↗
The Medicines Company (NASDAQ: MDCO) and Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) today announced new, positive data from the ORION-1 Phase II study of inclisiran, an investigational, first-in-class PCSK9 synthesis inhibitor being developed for the treatment of hypercholesterolemia. |
| 2017-03-17 | Alnylam Pharmaceuticals | Results The Medicines Company and Alnylam Pharmaceuticals Report Positive Final Results from ORION-1 Phase II Study of Inclisiran alnylam.com ↗
The Medicines Company (NASDAQ: MDCO) and Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) today announced positive final results from the ORION-1 Phase II study of inclisiran, an investigational, first-in-class PCSK9 synthesis inhibitor being developed for the treatment of hypercholesterolemia. |
| 2016-11-15 | Alnylam Pharmaceuticals | Results The Medicines Company and Alnylam Pharmaceuticals Present Positive Results from ORION-1 Phase 2 Study of Inclisiran (formerly PCSK9si) alnylam.com ↗
The Medicines Company (Nasdaq:MDCO) and Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY) today announced positive results from the analysis of Day 90 data for 497 patients, as well as analysis of preliminary Day 180 data for 189 patients, enrolled in the ORION -1 Phase 2 study of inclisiran. |
| 2016-11-13 | Alnylam Pharmaceuticals | Results Alnylam Pharmaceuticals and The Medicines Company Announce Publication of Phase 1 Clinical Data with Inclisiran (ALN- PCSsc) in the New England Journal of Medicine alnylam.com ↗
Results from the study showed doses ≥300 mg (single or multiple doses) significantly reduced PCSK9 and LDL cholesterol for at least 6 months. |
All press releases naming this drug 27 releases
Evidence & citations 9 cited values
Every value below carries the sentence it was read from. 59 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Inclisiran | “Patients randomized to the "PCSK9i early" intensified therapy group will receive initial treatment with a PCSK9 inhibitor-either evolocumab 140 mg or alirocumab 75 mg, both...” NCT06791031 ↗50“BACKGROUND: Inclisiran (ALN-PCSsc) is a long-acting RNA interference (RNAi) therapeutic agent that inhibits the synthesis of proprotein convertase subtilisin-kexin type 9...” PMID 27959715 ↗ Nov 2016 |
| Known as | ALN-PCSSC | “BACKGROUND: Inclisiran (ALN-PCSsc) is a long-acting RNA interference (RNAi) therapeutic agent that inhibits the synthesis of proprotein convertase subtilisin-kexin type 9...” PMID 27959715 ↗ Nov 2016 |
| Known as | inclisiran sodium | “Patients randomized to the "PCSK9i early" intensified therapy group will receive initial treatment with a PCSK9 inhibitor-either evolocumab 140 mg or alirocumab 75 mg, both...” NCT06791031 ↗16“Inclisiran (inclisiran sodium 300 mg subcutaneous (s.c.) for participants with body weight ≥23 kg and inclisiran sodium 180 mg s.c. for participants with body weight \<23 kg.” NCT06597019 ↗ “The ORION-18 was a phase 3 double-blind trial in which patients were randomized 1:1 to receive either 300 mg inclisiran sodium or matching placebo on days 1, 90, and 270.” PMID 38371290 ↗ Nov 2023 “Patients eligible for part 1 (double-blind, 6 months) were randomized 2:1 to receive either 300 mg of inclisiran sodium (equivalent to 284 mg of inclisiran) or placebo.” PMID 37850379 ↗ Oct 2023 “Patients will be treated with inclisiran sodium 300 mg or placebo, administered subcutaneously at Day 1, Day 90, and every 6 months thereafter.” PMID 42203164 ↗ May 2026 “The participants will receive Inclisiran sodium 300 mg subcutaneous at randomization (Day 1, Baseline visit) and Day 90” NCT07102628 ↗ “subcutaneous injections of inclisiran sodium (at a dose of 300 mg) or matching placebo on days 1, 90, 270, and 450.” PMID 32197277 ↗ Mar 2020 “Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL solution for subcutaneous injection” NCT04652726 ↗ “100 mg inclisiran sodium (equivalent to 94.5 mg inclisiran) in 0.5 mL solution.” NCT04774003 ↗ “Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL” NCT05739383 ↗ “Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL” NCT06865885 ↗ |
| Known as | KJX839 | “Inclisiran, also known as KJX839, is a medication made to reduce the level of "bad" cholesterol (LDL-cholesterol) in the blood.” NCT04807400 ↗ |
| Known as | Leqvio | “Inclisiran (Leqvio®) 300mg” NCT07421232 ↗ |
| Action | Inhibit | “inhibits the synthesis of proprotein convertase subtilisin-kexin type 9 (PCSK9)” PMID 27959715 ↗ Nov 2016 |
| Modality | Small interfering RNA (siRNA) | “long-acting RNA interference (RNAi) therapeutic agent” PMID 27959715 ↗ Nov 2016 |
| Route | Subcutaneous | “subcutaneous injection of inclisiran” PMID 27959715 ↗ Nov 2016 |
| Target | PCSK9 | “inhibits the synthesis of proprotein convertase subtilisin-kexin type 9 (PCSK9)” PMID 27959715 ↗ Nov 2016 |