Hereditary Transthyretin Amyloidosis (hATTR) - Pipeline Insight, 2024

This “Hereditary transthyretin amyloidosis - Pipeline Insight, 2024,” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Hereditary transthyretin amyloidosis pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Hereditary transthyretin amyloidosis Understanding

Hereditary transthyretin amyloidosis: Overview

Hereditary transthyretin (ATTR) amyloidosis is characterized by a slowly progressive peripheral sensorimotor and/or autonomic neuropathy as well as non-neuropathic changes of cardiomyopathy, nephropathy, vitreous opacities, and CNS amyloidosis. It is a rare inherited condition characterized by abnormal build-up of a protein called amyloid in the body’s organs and tissues. Symptoms start in adulthood and get worse over time. Signs and symptoms depend on where the amyloid protein is building up. Amyloid build-up in the nerves of the peripheral nervous system causes a loss of sensation in the lower limbs, feet, and hands (peripheral neuropathy). Amyloid build-up can also affect the involuntary body functions, such as blood pressure, heart rate, and digestion. Other areas of the body that may be affected are the heart, kidneys, eyes, and gastrointestinal tract. Disease heterogeneity and its rarity make a diagnosis of h ATTR amyloidosis challenging. Diagnosis can be confirmed via biopsy of the affected tissue or organ followed by staining with Congo red to confirm the presence of amyloid. Diagnosis can be established less invasively through biopsy of the salivary gland, an endoscopic biopsy of the gastric mucosa, or subcutaneous fat aspiration. Western blot analysis, immunohistochemical staining, laser microdissection, proteomics, and mass spectrometry are subsequently used to characterize amyloid type. Current treatment options for patients with TTR amyloidosis are limited, with only symptomatic treatment and transplantation. For patients diagnosed with TTR-FAP who have mild or moderate disease and confirmed by genetic testing and biopsy, a liver transplant is the current standard ofcare.

Hereditary transthyretin amyloidosis - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Hereditary transthyretin amyloidosis pipeline landscape is provided which includes the disease overview and Hereditary transthyretin amyloidosis treatment guidelines. The assessment part of the report embraces, in depth Hereditary transthyretin amyloidosis commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Hereditary transthyretin amyloidosis collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Hereditary transthyretin amyloidosis R&D. The therapies under development are focused on novel approaches to treat/improve Hereditary transthyretin amyloidosis.

Hereditary transthyretin amyloidosis Emerging Drugs Chapters

This segment of the Hereditary transthyretin amyloidosis report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Hereditary transthyretin amyloidosis Emerging Drugs

Vutrisiran: Alnylam Pharmaceuticals Vutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hereditary (h ATTR) and wild-type (wt ATTR) amyloidosis. It is designed to target and silence specific messenger RNA, blocking the production of wild-type and variant transthyretin (TTR) protein before it is made. Quarterly administration of vutrisiran may help to reduce deposition and facilitate the clearance of TTR amyloid deposits in tissues and potentially restore function to these tissues. Vutrisiran utilizes Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform, designed for increased potency and high metabolic stability that allows for infrequent subcutaneous injections. HELIOS-B is a randomized, double-blind, placebo-controlled Phase 3 study of ~600 adult patients with ATTR amyloidosis with cardiomyopathy (including both hATTR and wtATTR amyloidosis). The U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug Application (NDA) for vutrisiran, an investigational RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The FDA has set an action date of April 14, 2022 under the Prescription Drug User Fee Act (PDUFA), and the Agency has indicated that they are not currently planning an advisory committee meeting as part of theNDAreview.

AKCEA-TTR-LRx: Ionis Pharmaceuticals AKCEA-TTR-LRx is an antisense drug developed using Ionis’ proprietary LIgand Conjugated Antisense (LICA) technology platform and is designed to inhibit production of TTR. It was discovered by Ionis and is being co-developed by Ionis and Akcea. In a Phase 1 clinical trial, patients treated with AKCEA-TTR-LRx experienced reductions in TTR of up to 94 percent at the highest dose. CARDIO-TTRansform is a global, double-blind, randomized, placebo-controlled Phase III cardiovascular outcome study. It is designed to compare AKCEA-TTR-LRx to placebo in patients with both wild type and hereditary ATTR cardiomyopathy who are on the current available standard of care.

Hereditary transthyretin amyloidosis: Therapeutic Assessment

This segment of the report provides insights about the different Hereditary transthyretin amyloidosis drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Hereditary transthyretin amyloidosis

There are approx. 8+ key companies which are developing the therapies for Hereditary transthyretin amyloidosis. The companies which have their Hereditary transthyretin amyloidosis drug candidates in the most advanced stage, i.e. Preregistration include, Alnylam Pharmaceuticals.

Phases

This report covers around 8+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Hereditary transthyretin amyloidosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Parenteral
• Intravenous
• Subcutaneous
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Hereditary transthyretin amyloidosis: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Hereditary transthyretin amyloidosis therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Hereditary transthyretin amyloidosis drugs.

Hereditary transthyretin amyloidosis Report Insights

• Hereditary transthyretin amyloidosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Hereditary transthyretin amyloidosis Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Hereditary transthyretin amyloidosis drugs?
• How many Hereditary transthyretin amyloidosis drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Hereditary transthyretin amyloidosis?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Hereditary transthyretin amyloidosis therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Hereditary transthyretin amyloidosis and their status?
• What are the key designations that have been granted to the emerging drugs?

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