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The publisher's latest Pharmaceutical and Healthcare disease pipeline guide Familial Amyloid Neuropathies - Drugs In Development, 2022, provides an overview of the Familial Amyloid Neuropathies (Metabolic Disorders) pipeline landscape.Speak directly to the analyst to clarify any post sales queries you may have.
Familial amyloid neuropathy is a slowly progressive condition characterized by the buildup of abnormal deposits of a protein called amyloid (amyloidosis) in the body's organs and tissues. Symptoms include numbness, tingling, pins and needles in the feet and hands, weakness and pain in the arms and legs, loss of sensation, urinary retention, reduced sweating, ankle swelling, fatigue, nausea, weight loss, dizziness and fainting. Supportive treatment includes diuretics, calcium channel blockers, beta blockers, angiotensin receptor blockers and angiotensin converting enzyme (ACE) inhibitors.
Report Highlights
The publisher's Pharmaceutical and Healthcare latest pipeline guide Familial Amyloid Neuropathies - Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Familial Amyloid Neuropathies (Metabolic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.The Familial Amyloid Neuropathies (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Familial Amyloid Neuropathies and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies/Universities/Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 2, 1, 3, 1 and 1 respectively. Similarly, the Universities portfolio in Phase II and Preclinical stages comprises 1 and 1 molecules, respectively.
Familial Amyloid Neuropathies (Metabolic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from the publisher's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.
Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
Scope
- The pipeline guide provides a snapshot of the global therapeutic landscape of Familial Amyloid Neuropathies (Metabolic Disorders).
- The pipeline guide reviews pipeline therapeutics for Familial Amyloid Neuropathies (Metabolic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Familial Amyloid Neuropathies (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Familial Amyloid Neuropathies (Metabolic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Familial Amyloid Neuropathies (Metabolic Disorders)
Reasons to Buy
- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Familial Amyloid Neuropathies (Metabolic Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Familial Amyloid Neuropathies (Metabolic Disorders) pipeline depth and focus of Indication therapeutics.
- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.
Table of Contents
- Introduction
- 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 the Publisher
- Disclaimer
- 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
- 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
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- ADRx Inc
- Akcea Therapeutics Inc
- Alnylam Pharmaceuticals Inc
- BSIM Therapeutics SA
- Corino Therapeutics Inc
- Novo Nordisk AS
- Proclara Biosciences Inc
- Regeneron Pharmaceuticals Inc
