家族性アミロイドニューロパチーは、アミロイドと呼ばれるタンパク質の異常沈着（アミロイドーシス）が体内の臓器や組織に蓄積されることを特徴とする、ゆっくりと進行する疾患です。症状としては、足や手のしびれ、ピリピリ感、手足の脱力感や痛み、感覚の喪失、尿閉、発汗減少、足首のむくみ、疲労、吐き気、体重減少、めまい、失神などがあります。支持療法として、利尿薬、カルシウム拮抗薬、β遮断薬、アンジオテンシン受容体拮抗薬、アンジオテンシン変換酵素（ACE）阻害薬などが使用されます。
目次
Table of Contents
Table of Contents
List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Familial Amyloid Neuropathies - Overview
Familial Amyloid Neuropathies - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Familial Amyloid Neuropathies - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Familial Amyloid Neuropathies - Companies Involved in Therapeutics Development
ADRx Inc
Akcea Therapeutics Inc
Alnylam Pharmaceuticals Inc
BSIM Therapeutics SA
Corino Therapeutics Inc
Novo Nordisk AS
Proclara Biosciences Inc
Regeneron Pharmaceuticals Inc
Familial Amyloid Neuropathies - Drug Profiles
Antibody to Inhibit TTR for Familial Transthyretin Amyloidosis - Drug Profile
Product Description
Mechanism Of Action
CLR-01 - Drug Profile
Product Description
Mechanism Of Action
doxycycline hyclate - Drug Profile
Product Description
Mechanism Of Action
eplontersen sodium - Drug Profile
Product Description
Mechanism Of Action
NNC-6019 - Drug Profile
Product Description
Mechanism Of Action
NPT-189 - Drug Profile
Product Description
Mechanism Of Action
NTLA-2001 - Drug Profile
Product Description
Mechanism Of Action
patisiran - Drug Profile
Product Description
Mechanism Of Action
Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Polyneuropathy - Drug Profile
Product Description
Mechanism Of Action
tolcapone - Drug Profile
Product Description
Mechanism Of Action
vutrisiran - Drug Profile
Product Description
Mechanism Of Action
Familial Amyloid Neuropathies - Dormant Projects
Familial Amyloid Neuropathies - Discontinued Products
Familial Amyloid Neuropathies - Product Development Milestones
Featured News & Press Releases
Sep 07, 2022: Ionis presents positive results from phase 3 NEURO-TTRansform study at International Symposium on Amyloidosis
Jun 22, 2022: AstraZeneca-Ionis’ eplontersen meets endpoints in Phase III ATTRv-PN trial
Oct 27, 2021: Alnylam reports positive topline 18-month results from HELIOS-A phase 3 study of Vutrisiran in patients with hATTR amyloidosis with polyneuropathy
Jun 24, 2021: Alnylam announces U.S. Food and Drug Administration acceptance of new drug application for investigational Vutrisiran for the treatment of the polyneuropathy of hereditary ATTR amyloidosis
Nov 13, 2017: European Medicines Agency (EMA) Grants Alnylam Accelerated Assessment of Patisiran for Patients with Hereditary ATTR (hATTR) Amyloidosis
Nov 02, 2017: Sanofi and Alnylam Present Positive Complete Results from APOLLO Phase 3 study of Investigational Patisiran in Hereditary ATTR (hATTR) Amyloidosis Patients with Polyneuropathy
Sep 20, 2017: Arbutus’ LNP Licensee Alnylam Announces Positive Phase 3 Results for LNP-Enabled Patisiran Program
Sep 20, 2017: Sanofi and Alnylam Report Positive Topline Results from APOLLO Phase 3 Study of Patisiran in Hereditary ATTR (hATTR) Amyloidosis Patients with Polyneuropathy
Apr 26, 2017: 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
Oct 10, 2016: Data Monitoring Committee Recommends Continuation of APOLLO Phase 3 Clinical Trial of Patisiran for Hereditary ATTR Amyloidosis with Polyneuropathy (hATTR-PN)
Aug 16, 2016: BSIM Therapeutics was awarded research and development contract fund
Jul 01, 2016: Alnylam Reports New Results on patisiran at the XV International Symposium on Amyloidosis
Jun 27, 2016: Alnylam to Report New Patisiran Results at the XV International Symposium on Amyloidosis
Apr 20, 2016: 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)
Feb 01, 2016: Alnylam Completes Enrollment in APOLLO Phase 3 Study with Patisiran, an Investigational RNAi Therapeutic for Patients with Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List of Tables
Number of Products under Development for Familial Amyloid Neuropathies, 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Universities/Institutes, 2022
Products under Development by Companies, 2022
Products under Development by Universities/Institutes, 2022
Number of Products by Stage and Target, 2022
Number of Products by Stage and Mechanism of Action, 2022
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Familial Amyloid Neuropathies - Pipeline by ADRx Inc, 2022
Familial Amyloid Neuropathies - Pipeline by Akcea Therapeutics Inc, 2022
Familial Amyloid Neuropathies - Pipeline by Alnylam Pharmaceuticals Inc, 2022
Familial Amyloid Neuropathies - Pipeline by BSIM Therapeutics SA, 2022
Familial Amyloid Neuropathies - Pipeline by Corino Therapeutics Inc, 2022
Familial Amyloid Neuropathies - Pipeline by Novo Nordisk AS, 2022
Familial Amyloid Neuropathies - Pipeline by Proclara Biosciences Inc, 2022
Familial Amyloid Neuropathies - Pipeline by Regeneron Pharmaceuticals Inc, 2022
Familial Amyloid Neuropathies - Dormant Projects, 2022
Familial Amyloid Neuropathies - Discontinued Products, 2022
List of Figures
Number of Products under Development for Familial Amyloid Neuropathies, 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Universities/Institutes, 2022
Number of Products by Top 10 Targets, 2022
Number of Products by Stage and Top 10 Targets, 2022
Number of Products by Top 10 Mechanism of Actions, 2022
Number of Products by Stage and Top 10 Mechanism of Actions, 2022
Number of Products by Top 10 Routes of Administration, 2022
Number of Products by Stage and Top 10 Routes of Administration, 2022
Number of Products by Top 10 Molecule Types, 2022
Number of Products by Stage and Top 10 Molecule Types, 2022
