Drugs / Patisiran
Regulatory milestones approvals, filings & regulatory actions · 13 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed AH amyloidosis 4 milestones | |||||
| Rejected (CRL) | US (FDA) | — | AH amyloidosis | 2023-10-09 | “FDA has issued a Complete Response Letter (CRL) in response to the Company’s supplemental New Drug Application (sNDA) for patisiran” alnylam.com ↗ |
| Filed | Other | ONPATTRO | AH amyloidosis | 2023-05-04 | “Submitted an sNDA to the Brazilian Health Regulatory Agency (ANVISA) for ONPATTRO ( patisiran ) for the treatment of the cardiomyopathy of ATTR amyloidosis.” alnylam.com ↗ |
| Filed | US (FDA) | ONPATTRO | AH amyloidosis | 2022-12-08 | “today announced the submission of its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for patisiran” alnylam.com ↗ “sNDA for patisiran for the treatment of the cardiomyopathy of ATTR amyloidosis is under review by the U.S. Food and Drug Administration” alnylam.com ↗ |
| Filed | Other | — | AH amyloidosis | 2019-02-07 | “Continued global efforts to bring ONPATTRO to patients with filing of a New Drug Submission (NDS) in Canada” alnylam.com ↗ “a Marketing Authorisation Application (MAA) in Switzerland, which has now been accepted” alnylam.com ↗ |
| Filed ATTRV30M amyloidosis 1 milestone | |||||
| Filed | Other | ONPATTRO | ATTRV30M amyloidosis | 2019-10-10 | “patisiran for the treatment of patients with hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy has been filed with the Brazilian Health Regulatory...” alnylam.com ↗ “Filed a marketing authorization application with the Brazilian Health Regulatory Agency (ANVISA) for patisiran for the treatment of hATTR amyloidosis patients with polyneuropathy.” alnylam.com ↗ “Patisiran – which will be commercialized following regulatory approval under the brand name ONPATTRO ® – is Alnylam’s first investigational drug submitted for review in Brazil .” alnylam.com ↗ |
| Filed hereditary amyloidosis 5 milestones | |||||
| Filed | Japan (PMDA) | — | hereditary amyloidosis | 2018-09-28 | “submitted a New Drug Application (NDA) to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for approval of patisiran for the treatment of hereditary...” alnylam.com ↗ |
| Filed | US (FDA) | — | hereditary amyloidosis | 2018-05-03 | “Received acceptance from the United States Food and Drug Administration ( FDA ) and the European Medicines Agency (EMA) of patisiran’s New Drug Application (NDA)” alnylam.com ↗ |
| Filed | US (FDA) | — | hereditary amyloidosis | 2018-02-01 | “the U.S. Food and Drug Administration ( FDA ) has accepted for filing its New Drug Application (NDA) for patisiran” alnylam.com ↗ |
| Filed | EU (EMA) | — | hereditary amyloidosis | 2017-12-18 | “European Medicines Agency (EMA) has accepted the Marketing Authorisation Application (MAA) and initiated its review for patisiran” alnylam.com ↗ “submission of a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for patisiran” alnylam.com ↗ |
| Filed | US (FDA) | — | hereditary amyloidosis | 2017-11-16 | “completion of the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration ( FDA ) for patisiran” alnylam.com ↗ “initiated submission of a rolling New Drug Application (NDA) to the U.S. Food and Drug Administration ( FDA ) for patisiran” alnylam.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansion | US (FDA) | ONPATTRO | — | 2023-01-13 | fda.gov ↗ |
| Approved | EU (EMA) | Onpattro | — | 2018-08-27 | europa.eu ↗ |
| Approved | US (FDA) | ONPATTRO | — | 2018-08-10 | fda.gov ↗ |
Trials 8
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 01 trial | ||||||
| Phase 0 | NCT05023889 | Aug 2022 → Apr 2026 | polyneuropathy, wild type ATTR amyloidosis | Austin Neuromuscular Center | Completed | No outcome recorded |
| Phase 22 trials | ||||||
| Phase 2 | NCT01961921 | Oct 2013 → Jul 2016 | familial amyloid neuropathy | Alnylam Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT01617967 | May 2012 → Oct 2013 | familial amyloid neuropathy | Alnylam Pharmaceuticals | Completed | No outcome recorded |
| Phase 33 trials · 2 met primary | ||||||
| Phase 3 | NCT03997383 | Sep 2019 → Jun 2022 | — | Alnylam Pharmaceuticals | Completed | Met primary |
| Phase 3 | NCT03862807 | Mar 2019 → Oct 2020 | hereditary amyloidosis | Alnylam Pharmaceuticals | Completed | No outcome recorded |
| Phase 3 | NCT02510261 | Jul 2015 → Nov 2022 | amyloidosis | Alnylam Pharmaceuticals | Completed | Met primary |
| Phase not stated2 trials | ||||||
| — | NCT02939820 | — | familial amyloid neuropathy | Alnylam Pharmaceuticals | Approved for marketing | No outcome recorded |
| — | NCT05505838 | — | amyloidosis | Alnylam Pharmaceuticals | No longer available | No outcome recorded |
| Comparator or background therapy4 trials | ||||||
| Phase 1 | NCT02053454 Comparator | Jan → Mar 2014 | — | Alnylam Pharmaceuticals | Completed | No outcome recorded |
| Phase 1 | NCT01559077 Comparator | Mar → Jun 2012 | hereditary amyloidosis | Alnylam Pharmaceuticals | Completed | No outcome recorded |
| Phase 3 | NCT03759379 Comparator | Feb 2019 → Nov 2020 | hereditary amyloidosis | Alnylam Pharmaceuticals | Completed | Met primary |
| Phase 3 | NCT01960348 Comparator | Nov 2013 → Aug 2017 | familial amyloid neuropathy | Alnylam Pharmaceuticals | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 72
| Date | Issuer | Release |
|---|---|---|
| 2026-08-30 | Alnylam Pharmaceuticals | Results Alnylam Presents New Data at ESC Congress 2026 Reinforcing Strength in RNAi-Powered TTR Silencing Across ATTR-CM Patient Populations and Treatment Settings alnylam.com ↗
Alnylam Pharmaceuticals, Inc . (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced new data at the European Society of Cardiology (ESC) Congress 2026 demonstrating the strength of RNAi-powered silencing for cardiovascular disease. |
| 2026-05-12 | Alnylam Pharmaceuticals | Results New Data Analyses Presented at Heart Failure 2026 Demonstrate Robust and Consistent Clinical Benefit of Vutrisiran as a First-Line Treatment Option Across ATTR-CM Patient Populations, Including Patients with a High Disease Burden alnylam.com ↗
Rates of ocular adverse events potentially associated with vitamin A deficiency were low and comparable to placebo. |
| 2023-10-25 | Alnylam Pharmaceuticals | Results Alnylam Announces Publication of Results from APOLLO-B Phase 3 Study of Patisiran in Patients with the Cardiomyopathy of ATTR Amyloidosis in the New England Journal of Medicine alnylam.com ↗
The data reported in the APOLLO-B Phase 3 study publication demonstrate that patisiran, an RNAi therapeutic targeting transthyretin (TTR), preserved functional capacity and health status and quality of life compared with placebo at 12 months. |
| 2023-10-09 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces Receipt of Complete Response Letter from U.S. FDA for Supplemental New Drug Application for Patisiran for the Treatment of the Cardiomyopathy of ATTR Amyloidosis alnylam.com ↗
The CRL indicated that the clinical meaningfulness of patisiran’s treatment effects for the cardiomyopathy of ATTR amyloidosis had not been established, and therefore, the sNDA for patisiran could not be approved in its present form. |
| 2023-09-13 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces Positive Outcome of FDA Advisory Committee Meeting on Patisiran for the Treatment of the Cardiomyopathy of ATTR Amyloidosis alnylam.com ↗
The CRDAC voted 9:3 that the benefits of patisiran outweigh its risks for the treatment of the cardiomyopathy of ATTR amyloidosis. |
| 2023-09-13 | Alnylam Pharmaceuticals | Regulatory Alnylam Stock Trading Halted Today; FDA Advisory Committee to Review Supplemental New Drug Application for Patisiran for the Treatment of the Cardiomyopathy of ATTR Amyloidosis alnylam.com ↗
The U.S. Food and Drug Administration’s (FDA) Cardiovascular and Renal Drugs Advisory Committee is meeting today to review the supplemental New Drug Application for patisiran, an investigational RNAi therapeutic in development for the treatment of the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis. |
| 2023-08-03 | Alnylam Pharmaceuticals | Regulatory Alnylam Pharmaceuticals Reports Second Quarter 2023 Financial Results and Highlights Recent Period Activity alnylam.com ↗
Submitted 18-Month APOLLO-B Data to the U.S. Food and Drug Administration as Amendment to Supplemental New Drug Application for Patisiran |
| 2023-06-30 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces Date of Planned FDA Advisory Committee Meeting for ONPATTRO® (patisiran) for the Treatment of the Cardiomyopathy of ATTR Amyloidosis alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has set a date of September 13, 2023 for the meeting of the Cardiovascular and Renal Drugs Advisory Committee to review the supplemental New Drug Application for patisiran, an investigational treatment for the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis. |
| 2023-05-20 | Alnylam Pharmaceuticals | Results Alnylam Presents 18-Month Results from the APOLLO-B Phase 3 Study of Patisiran in Patients with ATTR Amyloidosis with Cardiomyopathy alnylam.com ↗
The results were presented at the Annual Congress of the Heart Failure Association of the European Society of Cardiology (Heart Failure 2023; May 20-23, 2023 ). |
| 2023-02-23 | Alnylam Pharmaceuticals | Regulatory Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2022 Financial Results and Highlights Recent Period Activity alnylam.com ↗
Submitted and received acceptance of the sNDA for ONPATTRO (patisiran) for the treatment of the cardiomyopathy of ATTR amyloidosis. |
| 2023-02-21 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces U.S. Food and Drug Administration (FDA) Acceptance of Supplemental New Drug Application for ONPATTRO® (patisiran) for the Treatment of the Cardiomyopathy of ATTR Amyloidosis alnylam.com ↗
today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the Company’s supplemental New Drug Application (sNDA) for patisiran, an investigational RNAi therapeutic in development for the treatment of the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis. |
| 2022-12-08 | Alnylam Pharmaceuticals | Regulatory Alnylam Submits Supplemental New Drug Application (sNDA) to U.S. Food and Drug Administration (FDA) for ONPATTRO® (patisiran) for the Treatment of the Cardiomyopathy of ATTR Amyloidosis alnylam.com ↗
We are excited by today’s announcement as the submission of our sNDA brings us one step closer to making patisiran available to patients with ATTR amyloidosis with cardiomyopathy. |
| 2022-09-30 | Alnylam Pharmaceuticals | Results Alnylam Presents Additional Results from the APOLLO-B Phase 3 Study of Patisiran in Patients with ATTR Amyloidosis with Cardiomyopathy at Heart Failure Society of America Annual Meeting alnylam.com ↗ |
| 2022-09-08 | Alnylam Pharmaceuticals | Results Alnylam Presents Positive Results from the APOLLO-B Phase 3 Study of Patisiran in Patients with ATTR Amyloidosis with Cardiomyopathy alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced positive results from the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy. |
| 2022-08-03 | Alnylam Pharmaceuticals | Results Alnylam Reports Positive Topline Results from APOLLO-B Phase 3 Study of Patisiran in Patients with ATTR Amyloidosis with Cardiomyopathy alnylam.com ↗
The study also met the first secondary endpoint of change from baseline in quality of life compared to placebo, as measured by the Kansas City Cardiomyopathy Questionnaire (KCCQ) (p-value 0.0397). |
| 2021-06-07 | Alnylam Pharmaceuticals | Results Alnylam Announces New Data from ATTR Amyloidosis Programs at the Peripheral Nerve Society’s 2021 Annual Meeting alnylam.com ↗
In patients treated with patisiran, the median reduction in serum TTR levels compared to baseline was 91 percent, measured as an average of the month six and month 12 reduction. |
| 2020-09-03 | Alnylam Pharmaceuticals | Results Alnylam Highlights Prospects for Building an Industry-Leading ATTR Amyloidosis Franchise at RNAi Roundtable alnylam.com ↗
These data, presented at the European Society of Cardiology (ESC) 2020 Congress , suggest that treatment with patisiran may lead to cardiac amyloid regression in patients with cardiomyopathy associated with ATTR amyloidosis. |
| 2020-05-22 | Alnylam Pharmaceuticals | Results Alnylam Presents New Long-Term Results from Global Open-Label Extension Study of ONPATTRO® (patisiran) at the European Academy of Neurology Virtual Congress 2020 alnylam.com ↗
In addition, interim results were presented from a Phase 3b trial evaluating treatment with patisiran in patients with hATTR amyloidosis with disease progression after receiving an orthotopic liver transplant (post-OLT). |
| 2020-02-26 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces Approval in Brazil of ONPATTRO® for the Treatment of Hereditary ATTR Amyloidosis with Polyneuropathy alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the Brazilian Health Regulatory Agency (ANVISA) has approved ONPATTRO ® (patisiran) for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults with stage 1 or stage 2 polyneuropathy. |
| 2019-10-10 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces Filing for Marketing Authorization of ONPATTRO® (patisiran) in Brazil for the Treatment of Hereditary ATTR Amyloidosis with Polyneuropathy alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the marketing authorization application of patisiran for the treatment of patients with hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy has been filed with the Brazilian Health Regulatory Agency (ANVISA). |
| 2019-09-03 | Alnylam Pharmaceuticals | Results Alnylam Presents New Clinical Research Findings at the Second European Meeting of ATTR Amyloidosis for Doctors and Patients alnylam.com ↗
The Company and collaborators presented results from a proteome-wide biomarker analysis of samples from the APOLLO Phase 3 study of ONPATTRO ® (patisiran), an RNAi therapeutic for the treatment of the polyneuropathy of hereditary ATTR (hATTR) amyloidosis in adults. |
| 2019-07-23 | Alnylam Pharmaceuticals | Regulatory Alnylam Launches ONPATTRO® (patisiran) for the Treatment of Polyneuropathy in hATTR Amyloidosis, the First-Ever RNAi Therapeutic Approved in Canada alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNA interference (RNAi) therapeutics company, today announced the Health Canada approval and immediate availability of ONPATTRO ® (patisiran) for the treatment of polyneuropathy in adult patients with hereditary transthyretin-mediated (hATTR) amyloidosis 3 . |
| 2019-07-09 | Alnylam Pharmaceuticals | Regulatory First-in-class ‘gene-silencing’ therapy approved for NHS use in England to treat hereditary form of amyloidosis alnylam.com ↗
NICE has recommended patisiran to treat hereditary transthyretin-mediated amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy |
| 2019-06-24 | Alnylam Pharmaceuticals | Results Alnylam Presents New ONPATTRO® (patisiran) Results at the 2019 Peripheral Nerve Society Annual Meeting alnylam.com ↗ |
| 2019-06-18 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces Approval in Japan of ONPATTRO® for the Treatment of Hereditary ATTR Amyloidosis with Polyneuropathy alnylam.com ↗ |
| 2019-06-13 | Alnylam Pharmaceuticals | Results Alnylam to Report New Clinical Results for ONPATTRO® (patisiran) at Peripheral Nerve Society (PNS) Annual Meeting and the 5th Congress of the European Academy of Neurology (EAN) alnylam.com ↗ |
| 2018-10-02 | Alnylam Pharmaceuticals | Regulatory Alnylam Launches ONPATTRO™ (patisiran), the First-Ever RNAi Therapeutic, in Germany alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNA interference (RNAi) therapeutics company, today announced that ONPATTRO (patisiran) is now available in Germany . |
| 2018-09-28 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces Submission of New Drug Application in Japan for ONPATTRO™ (patisiran sodium) for Treatment of Hereditary ATTR Amyloidosis alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that it has submitted a New Drug Application (NDA) to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for approval of patisiran for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis. |
| 2018-09-24 | Alnylam Pharmaceuticals | Regulatory Alnylam Pharmaceuticals Receives Priority Review in Canada for Patisiran, an Investigational RNAi Therapeutic for the Treatment of Hereditary Transthyretin-mediated Amyloidosis (hATTR amyloidosis) with Polyneuropathy alnylam.com ↗ |
| 2018-09-14 | Alnylam Pharmaceuticals | Results Alnylam Announces Publication in Circulation of Exploratory Cardiac Endpoint Data from APOLLO Phase 3 Study of Patisiran alnylam.com ↗
The results were published online today in the journal Circulation, and showed that patisiran improved markers of cardiomyopathy in patients with hATTR amyloidosis with polyneuropathy. |
| 2018-08-30 | Alnylam Pharmaceuticals | Regulatory Alnylam Receives Approval of ONPATTRO™ (patisiran) in Europe alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the European Commission (EC) has granted marketing authorization for ONPATTRO ™ (patisiran) |
| 2018-08-10 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces First-Ever FDA Approval of an RNAi Therapeutic, ONPATTRO™ (patisiran) for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the United States Food and Drug Administration ( FDA ) approved ONPATTRO™ (patisiran) lipid complex injection, a first-of-its-kind RNA interference (RNAi) therapeutic, for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. |
| 2018-08-03 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces that the United Kingdom’s MHRA Grants Early Access to Patisiran alnylam.com ↗
With this decision, eligible adults in the UK with hATTR amyloidosis can gain access to patisiran before the drug is granted marketing authorization by the European Commission (EC). |
| 2018-08-02 | Alnylam Pharmaceuticals | Regulatory Alnylam Pharmaceuticals Reports Second Quarter 2018 Financial Results and Highlights Recent Period Activity alnylam.com ↗
Received a positive opinion from CHMP recommending marketing authorization of ONPATTRO (patisiran) – a first-of-its-kind RNAi therapeutic – for the treatment of hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy. |
| 2018-07-27 | Alnylam Pharmaceuticals | Regulatory Alnylam Receives Positive CHMP Opinion for ONPATTRO™ (patisiran) for the Treatment of Hereditary Transthyretin-Mediated Amyloidosis in Adults with Stage 1 or Stage 2 Polyneuropathy alnylam.com ↗
The CHMP positive opinion is based on the evaluation of the effects of patisiran in patients with hATTR amyloidosis and its safety profile as demonstrated in the APOLLO Phase 3 study. |
| 2018-07-23 | Alnylam Pharmaceuticals | Results Alnylam Presents New Analyses of Clinical Results from APOLLO Phase 3 Study of Patisiran at 2018 Peripheral Nerve Society Annual Meeting alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company, announced today that the Company presented new analyses from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis, in six presentations at the 2018 Peripheral Nerve Society (PNS) Annual Meeting being held July 22-25 , in Baltimore, MD . |
| 2018-07-04 | Alnylam Pharmaceuticals | Results Alnylam Announces Publication of APOLLO Phase 3 Clinical Study Results for Investigational RNAi Therapeutic Patisiran in The New England Journal of Medicine alnylam.com ↗ |
| 2018-04-24 | Alnylam Pharmaceuticals | Results Alnylam Reports New Clinical Results from the APOLLO Phase 3 Study of Patisiran at the American Academy of Neurology 2018 Annual Meeting alnylam.com ↗
To that end, we are pleased to share these new results from the APOLLO study, including a post-hoc, exploratory analysis demonstrating a significant decrease in the composite rate of all-cause hospitalization and mortality in patients receiving patisiran compared to placebo. |
| 2018-03-28 | Alnylam Pharmaceuticals | Results Alnylam Presents New Clinical Results from the APOLLO Phase 3 Study of Patisiran at the 16th International Symposium on Amyloidosis alnylam.com ↗
The clinical results presented further highlight the robust profile of patisiran and provide evidence supporting patisiran as a potentially transformative treatment approach for patients with hATTR amyloidosis. |
| 2018-02-01 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces FDA Acceptance of New Drug Application (NDA) and Priority Review Status for Patisiran, an Investigational RNAi Therapeutic for the Treatment of Hereditary ATTR (hATTR) Amyloidosis alnylam.com ↗ |
| 2018-01-25 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces EMA Acceptance of Marketing Authorisation Application (MAA) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis alnylam.com ↗ |
| 2017-12-18 | Alnylam Pharmaceuticals | Regulatory Alnylam and Sanofi Submit Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis alnylam.com ↗ |
| 2017-12-12 | Alnylam Pharmaceuticals | Regulatory Alnylam Completes Submission of New Drug Application to U.S. Food and Drug Administration (FDA) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis alnylam.com ↗ |
| 2017-12-11 | Alnylam Pharmaceuticals | Regulatory Alnylam Announces Expansion of U.S. Orphan Drug Designation for Patisiran to Treatment of Transthyretin-Mediated Amyloidosis alnylam.com ↗ |
| 2017-11-20 | Alnylam Pharmaceuticals | Regulatory U.S. Food and Drug Administration (FDA) Grants Alnylam Breakthrough Therapy Designation (BTD) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis with Polyneuropathy alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the U.S. Food and Drug Administration ( FDA ) has granted Breakthrough Therapy Designation (BTD) for patisiran |
| 2017-11-16 | Alnylam Pharmaceuticals | Regulatory Alnylam Initiates Rolling Submission of New Drug Application (NDA) to U.S. Food and Drug Administration (FDA) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis alnylam.com ↗ |
| 2017-11-13 | Alnylam Pharmaceuticals | Regulatory European Medicines Agency (EMA) Grants Alnylam Accelerated Assessment of Patisiran for Patients with Hereditary ATTR (hATTR) Amyloidosis alnylam.com ↗ |
| 2017-11-02 | Alnylam Pharmaceuticals | Results Alnylam and Sanofi Present Positive Complete Results from APOLLO Phase 3 Study of Investigational Patisiran in Hereditary ATTR (hATTR) Amyloidosis Patients with Polyneuropathy alnylam.com ↗
The full APOLLO results showed improvement with patisiran relative to placebo in the primary endpoint of modified Neuropathy Impairment Score +7 (mNIS+7) and additional secondary endpoints |
| 2017-10-20 | Alnylam Pharmaceuticals | Results Alnylam and Collaborators to Present APOLLO Phase 3 Study Results at 1st European ATTR Amyloidosis Meeting for Patients and Doctors alnylam.com ↗ |
| 2017-09-20 | Alnylam Pharmaceuticals | Results Alnylam and Sanofi Report Positive Topline Results from APOLLO Phase 3 Study of Patisiran in Hereditary ATTR (hATTR) Amyloidosis Patients with Polyneuropathy alnylam.com ↗ |
| 2017-04-26 | Alnylam Pharmaceuticals | Results Alnylam Reports Final 24-Month Results from Phase 2 Open-Label Extension Study of Patisiran, an Investigational RNAi Therapeutic in Development for the Treatment of Hereditary ATTR (hATTR) Amyloidosis alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today final 24-month results from its Phase 2 open-label extension (OLE) study of patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of hATTR amyloidosis. |
| 2016-07-01 | Alnylam Pharmaceuticals | Results Alnylam Reports New Results from Investigational RNAi Therapeutic Programs for Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy (hATTR-PN) and Cardiomyopathy (hATTR-CM) alnylam.com ↗
At 24 Months, Patisiran Shows a Mean 6.7 Point Decrease in Modified Neuropathy Impairment Score (mNIS+7), Comparing Favorably with an Expected Mean 26-30 Point Increase Estimated from Historical Data |
| 2016-06-27 | Alnylam Pharmaceuticals | Results Alnylam to Report New Patisiran and Revusiran Results at the XV International Symposium on Amyloidosis alnylam.com ↗
All posters by Alnylam scientists and collaborators will be presented during the poster session on July 4, 2016 . |
| 2016-04-20 | Alnylam Pharmaceuticals | Results Alnylam Reports Complete 18-Month Data from Ongoing Phase 2 Open-Label Extension Study of Patisiran, an Investigational RNAi Therapeutic Targeting Transthyretin for the Treatment of Hereditary ATTR Amyloidosis with Polyneuropathy (hATTR-PN) alnylam.com ↗ |
| 2015-11-03 | Alnylam Pharmaceuticals | Results Alnylam Reports Positive Results from Ongoing Phase 2 Open-Label Extension (OLE) Studies for Patisiran and Revusiran, in Development for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis) alnylam.com ↗
Data from the patisiran Phase 2 OLE study provided continued evidence following 18 months of dosing that patisiran has the potential to halt neuropathy progression in patients with Familial Amyloidotic Polyneuropathy (FAP). |
| 2015-09-28 | Alnylam Pharmaceuticals | Results Alnylam and Collaborators Report First-Ever Evidence that Patisiran Reduces Pathogenic, Misfolded Transthyretin (TTR) Monomers and Oligomers in TTR-Mediated Amyloidosis (ATTR Amyloidosis) Patients with Familial Amyloidotic Polyneuropathy (FAP) alnylam.com ↗
Specifically, patisiran administration resulted in a rapid and sustained reduction of approximately 90% in serum non-native conformations of TTR (NNTTR). |
| 2015-04-21 | Alnylam Pharmaceuticals | Results Alnylam Reports 12-Month Clinical Data from Phase 2 Open Label Extension (OLE) Study of Patisiran, an Investigational RNAi Therapeutic for Patients with Familial Amyloidotic Polyneuropathy (FAP) alnylam.com ↗
Study results showed a mean 2.5 point decrease in modified Neuropathy Impairment Score (mNIS+7) at 12 months in patients who had reached the 12-month endpoint (N=20) at the time of data cutoff. |
| 2014-10-13 | Alnylam Pharmaceuticals | Results Alnylam Reports Six-Month Clinical Data from Patisiran Phase 2 Open-Label Extension (OLE) Study in Patients with Familial Amyloidotic Polyneuropathy (FAP) alnylam.com ↗
Results showed a mean 0.95 point decrease in modified Neuropathy Impairment Score (mNIS+7) at six months in 19 patients with mNIS+7 data available for the current analysis. |
| 2014-04-29 | Alnylam Pharmaceuticals | Results Alnylam Presents New Pre-clinical Data on RNAi Therapeutics Targeting Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis (ATTR) alnylam.com ↗
Alnylam Pharmaceuticals, Inc . (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today the presentation of new pre-clinical data on RNAi therapeutics targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR). |
| 2014-04-28 | Alnylam Pharmaceuticals | Results Alnylam and Collaborators Present New Clinical Data for Patisiran, an RNAi Therapeutic Targeting Transthyretin (TTR) in Development for the Treatment of TTR-Mediated Amyloidosis (ATTR) alnylam.com ↗
Preliminary results from the OLE study showed that multiple doses of patisiran achieved sustained knockdown of serum TTR protein levels at the 80% target level through 168 days. |
| 2013-11-25 | Alnylam Pharmaceuticals | Results Alnylam Earns $7 Million Milestone Payment from Genzyme for Phase II Success with Patisiran (ALN-TTR02), an RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis (ATTR) alnylam.com ↗
Results showed that multiple doses of patisiran led to robust and statistically significant knockdown of serum TTR protein levels of up to 96%, with mean levels of TTR knockdown exceeding 85%. |
| 2013-11-11 | Alnylam Pharmaceuticals | Regulatory Alnylam Receives Fast Track Designation for Patisiran (ALN-TTR02), an RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis (ATTR) alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to patisiran (ALN-TTR02) for the treatment of transthyretin (TTR)-familial amyloid polyneuropathy (FAP). |
| 2013-11-10 | Alnylam Pharmaceuticals | Results Alnylam Reports Positive Phase II Data for Patisiran (ALN-TTR02), an RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis (ATTR), and Initiates Phase III Trial alnylam.com ↗
Results showed that multiple doses of patisiran led to robust and statistically significant knockdown of serum TTR protein levels of up to 96%, with mean levels of TTR knockdown exceeding 85%. |
| 2013-09-30 | Alnylam Pharmaceuticals | Results Alnylam Completes Enrollment of Phase II Clinical Trial and Initiates Open-Label Extension (OLE) Study with ALN-TTR02, an RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of Transthyretin-Mediated Amyloidosis (ATTR) alnylam.com ↗
Recent interim results from this Phase II study showed that ALN-TTR02 achieved up to 93% knockdown of TTR — the disease-causing protein in ATTR. |
| 2013-08-28 | Alnylam Pharmaceuticals | Results Alnylam and Collaborators Publish Clinical Trial Results with RNAi Therapeutics Targeting Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis (ATTR) in the New England Journal of Medicine alnylam.com ↗
Alnylam Pharmaceuticals, Inc . (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today the publication of complete study results from Phase I trials with ALN-TTR01 and ALN-TTR02 in the New England Journal of Medicine . |
| 2013-06-30 | Alnylam Pharmaceuticals | Results Alnylam Reports Positive Phase II Data for ALN-TTR02, an RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis (ATTR) alnylam.com ↗
Interim results show that multiple doses of ALN-TTR02 led to robust and statistically significant (p < 0.001) knockdown of serum TTR protein levels of up to 93%. |
| 2012-07-16 | Alnylam Pharmaceuticals | Results Alnylam Reports Positive ALN-TTR02 Clinical Data, with Robust Knockdown of Serum Transthyretin (TTR) of up to 94% After Single Dose alnylam.com ↗
Results from this study show that administration of ALN-TTR02 leads to robust knockdown of serum TTR protein levels of up to 94%; the overall results were highly significant (p<0.00001 by ANOVA). |
| 2012-06-19 | Alnylam Pharmaceuticals | Regulatory Alnylam Receives Orphan Drug Designation from U.S. Food & Drug Administration for ALN-TTR02, an RNAi Therapeutic for the Treatment of Transthyretin-Mediated Amyloidosis (ATTR) alnylam.com ↗ |
| 2012-01-30 | Alnylam Pharmaceuticals | Regulatory Alnylam Receives Notice of Allowance from United States Patent and Trademark Office (USPTO) for New Patent Broadly Covering Second Generation Lipid Nanoparticles (LNP) for Systemic Delivery of RNAi Therapeutics alnylam.com ↗ |
| 2012-01-06 | Alnylam Pharmaceuticals | Regulatory Alnylam Files Clinical Trial Application for ALN-TTR02, an RNAi Therapeutic Targeting Transthyretin for the Treatment of Transthyretin-Mediated Amyloidosis alnylam.com ↗
Alnylam Files Clinical Trial Application for ALN-TTR02, an RNAi Therapeutic Targeting Transthyretin for the Treatment of Transthyretin-Mediated Amyloidosis |
| 2010-04-20 | Alnylam Pharmaceuticals | Results Alnylam and Collaborators Demonstrate Regression of Pathogenic Transthyretin (TTR) Amyloid Deposits Following Treatment with an RNAi Therapeutic in an Animal Model of TTR-Mediated Amyloidosis (ATTR) alnylam.com ↗
These new pre-clinical data demonstrate - for the first time - that treatment with an RNAi therapeutic can result in regression of pre-existing pathogenic TTR deposits in peripheral tissues. |
| 2010-01-19 | Alnylam Pharmaceuticals | Results Alnylam and Collaborators Present Data from Multiple Pre-Clinical and Clinical Programs at RNAi Keystone Symposium alnylam.com ↗
Alnylam and its collaborators presented data from Alnylam's therapeutic programs including transthyretin (TTR)-mediated amyloidosis and Huntington's disease, as well as new data on delivery approaches for the systemic delivery of RNAi therapeutics. |
All press releases naming this drug 90 releases
Evidence & citations 9 cited values
Every value below carries the sentence it was read from. 15 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | patisiran | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02939820 ↗ |
| Known as | ALN-18328 | ChEMBL registry synonym — accepted as the source's own label CHEMBL3989987 ↗ |
| Known as | ALN-TTR02 | “The purpose of this study is to evaluate the safety and tolerability of long-term dosing with ALN-TTR02 (patisiran) in patients with transthyretin (TTR) mediated amyloidosis (ATTR).” NCT01961921 ↗4“The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of patisiran (ALN-TTR02) in Japanese subjects” NCT02053454 ↗ “patisiran (ALN-TTR02) administered by intravenous (IV) infusion” NCT02939820 ↗ “patisiran (ALN-TTR02)” NCT01960348 ↗ |
| Known as | Genz-438027 | ChEMBL registry synonym — accepted as the source's own label CHEMBL3989987 ↗ |
| Known as | SAR-438037 | ChEMBL registry synonym — accepted as the source's own label CHEMBL3989987 ↗ |
| Action | Inhibit | “Administration of patisiran led to rapid, dose-dependent, and durable knockdown of transthyretin” PMID 26338094 ↗ Sep 2015 |
| Modality | Small interfering RNA (siRNA) | “We identified a potent antitransthyretin small interfering RNA, which was encapsulated in two distinct first- and second-generation formulations of lipid nanoparticles,...” PMID 23984729 ↗ Aug 2013 |
| Route | Intravenous | “patients with FAP were administered 2 intravenous infusions of patisiran” PMID 26338094 ↗ Sep 2015 |
| Target | TTR | “Administration of patisiran led to rapid, dose-dependent, and durable knockdown of transthyretin” PMID 26338094 ↗ Sep 2015 |