Onpattro

(Patisiran)

ApprovedRNA therapy

Onpattro is a rna therapy that works as a TTR Sirna.[2] It is being used across 4 indications, with its most advanced program in Approved.[1]

Onpattro is one of 73 sirna programs targeting Transthyretin Amyloid Cardiomyopathy. It is currently in Approved.

Indications

Total Trials

Active Trials

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

Transthyretin Amyloid CardiomyopathyActive, not recruiting

PolyneuropathyActive, not recruiting

Heart FailureApproved

CardiomyopathyActive, not recruiting

Transthyretin Amyloid CardiomyopathyApproved

Active, not recruiting

PolyneuropathyPreclinical

Active, not recruiting

Heart FailureApproved

Approved

CardiomyopathyPhase 3

Active, not recruiting

Competition

Competitive Landscape

206 competitors in Heart Failure

#DrugCompanyPhaseTrials

Onpattro

Alnylam Pharmaceuticals

Approved

11 2

Bayer Aspirin

Bayer

Preclinical

2 3

Medrol

Pfizer

Preclinical

1 4

Amidate

Pfizer

Preclinical

1 5

Activase

Xylocaine

1 7

Potassium Iodide

MMeda Pharmaceuticals

Preclinical

1 8

Oraverse

LLife Extension Foundation Inc.

Preclinical

1 9

Statin

Bristol-Myers Squibb

Preclinical

1 10

Optimal Medical Therapy

HHelios Health Institute GmbH

Preclinical

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q1 2027

Phase 3Active, not recruiting

Cardiomyopathy

NCT03997383

Clinical

Trials by Indication[1]

Amyloidosis5 trials

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

Completed· Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals·Phase 3·NCT01960348·225 enrolled

Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)

Approved for marketing· Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals··NCT02939820

The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)

Completed· Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals·Phase 3·NCT02510261·211 enrolled

Show 2 more trials

Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy

Active, not recruiting·AAustin Neuromuscular Center·Phase 1·NCT05023889·10 enrolled

Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis

Completed· Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals·Phase 2·NCT01617967·29 enrolled

Cardiomyopathy2 trials

Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy

No longer available· Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals··NCT05505838

APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

Active, not recruiting· Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals·Phase 3·NCT03997383·360 enrolled

Tics1 trial

A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers

Completed· Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals·Phase 1·NCT02053454·12 enrolled

Polyneuropathy1 trial

A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

Completed· Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals··NCT04201418·67 enrolled

Hereditary Transthyretin Amyloidosis1 trial

HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

Completed· Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals·Phase 3·NCT03759379·164 enrolled

1 more indication

Liver Transplant1 trial

Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant

Completed· Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals·Phase 3·NCT03862807·24 enrolled

Regulatory

FDA Approval Timeline[4]

Aug 10, 2018

ONPATTRO

NDA NDA210922

Aug 10, 2018

ONPATTRO

NDA NDA210922

Sep 16, 2019

ONPATTRO

NDA NDA210922

Apr 17, 2020

ONPATTRO

NDA NDA210922

May 6, 2021

ONPATTRO

NDA NDA210922

Oct 3, 2022

ONPATTRO

NDA NDA210922

Jan 13, 2023

ONPATTRO

NDA NDA210922

Sources

References

[1]
ClinicalTrials.gov — 11 trial records for Patisiran. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Patisiran compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Patisiran. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA210922). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Onpattro. U.S. Food & Drug Administration.
[6]
PubMed — 1 publication indexed for Patisiran. U.S. National Library of Medicine.
[7]
SEC EDGAR — Alnylam Pharmaceuticals company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: TTR Sirna[2]
Modality: RNA therapy[2]
Highest Phase: Approved[1]
Company: Alnylam Pharmaceuticals

Patent Landscape[5]

Patent Cliff

Oct 3, 2027

Earliest patent expiration — generic entry possible after this date

Patent #Expires

8642076Oct 3, 2027

11141378Apr 15, 2029

8058069Apr 15, 2029

8492359Apr 15, 2029

9364435Apr 15, 2029

8 more patents

8822668Apr 15, 2029

8741866Oct 20, 2029

9234196Oct 20, 2029

10240152Oct 20, 2029

8802644Oct 21, 2030

8158601Nov 10, 2030

8168775Aug 10, 2032

11079379Aug 27, 2035

Key Publications[6]

Patisiran in ATTR Cardiomyopathy: APOLLO-B Phase 3 Results

N Engl J MedJul 6, 2023DOI: 10.1056/NEJMoa2305862

Onpattro

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q1 2027

Trials by Indication[1]

Amyloidosis5 trials

Cardiomyopathy2 trials

Tics1 trial

Polyneuropathy1 trial

Hereditary Transthyretin Amyloidosis1 trial

Liver Transplant1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

Key Publications[6]

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