Therapeutic Market Overview for Systemic Amyloidosis: Key Trends and Major Player Analysis

The market for systemic amyloidosis therapeutics is set to experience significant growth over the coming years, driven by advancements in treatment options and increasing diagnosis rates. This expanding market is attracting considerable attention as innovative therapies continue to emerge, promising better patient outcomes for this rare and complex disease.

Systemic Amyloidosis Therapeutic Market Size and Forecast to 2030
The systemic amyloidosis therapeutic market is projected to grow steadily, reaching a valuation of $8.88 billion by 2030. This expansion reflects a compound annual growth rate (CAGR) of 9.4%, driven by factors such as improved identification and diagnosis of rare diseases, the broadening availability of targeted biologic drugs, more approvals for gene silencing therapies, the growth of specialty clinic networks, and enhanced incentives for orphan drugs. Key trends expected to shape this market include the development of amyloid protein stabilizers, gene silencing therapies tailored for amyloidosis, monoclonal antibody treatments, combination therapy protocols, and organ-specific supportive drug strategies.

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Leading Organizations in the Systemic Amyloidosis Therapeutic Industry
Several prominent companies are spearheading innovation and development in the systemic amyloidosis therapeutic space. These include Johnson & Johnson Services Inc., Sanofi SA, Novartis Plc, AstraZeneca Plc, GSK Plc, Novo Nordisk A/S, Takeda Pharmaceutical Company Limited, Regeneron Pharmaceuticals Inc., Alnylam Pharmaceuticals Inc., Ionis Pharmaceuticals, Arcturus Therapeutics Inc., Prothena Biosciences, Fortress Biotech Inc., Intellia Therapeutics Inc., Zentalis Pharmaceuticals Inc., BridgeBio Pharma Inc., Neurimmune AG, HaemaLogix Pty Ltd., Attralus Inc., and Oncopeptides AB.

Recent Collaborations Advancing Treatment Options
In April 2025, Neurimmune, a clinical-stage biopharmaceutical company based in Switzerland and specializing in protein aggregation diseases, entered a partnership with Alexion, AstraZeneca Rare Disease. The collaboration aims to develop and commercialize NI009, a monoclonal antibody designed to clear amyloid deposits in patients with light chain (AL) amyloidosis. This partnership seeks to accelerate the availability of fibril-depleting therapies that specifically target lambda light chain amyloid accumulation. Alexion is a US-based global biopharmaceutical firm focused on late-stage development, manufacturing, and commercialization of treatments for serious systemic disorders, including amyloidosis.

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Emerging Treatment Approaches in the Systemic Amyloidosis Therapeutic Market
Several industry leaders are focusing on creating advanced targeted therapies that address the genetic basis of protein misfolding diseases. RNA interference (RNAi)-based treatments are increasingly prominent, designed to silence disease-causing genes and reduce the production of harmful proteins that lead to systemic organ damage. This genetic approach aims to slow disease progression and enhance long-term patient well-being.

A notable example is the launch of AMVUTTRA by Alnylam Pharmaceuticals in March 2025. This RNAi therapeutic targets transthyretin amyloidosis with cardiomyopathy (ATTR-CM) by reducing transthyretin production at its source. AMVUTTRA is the first RNAi treatment proven to lower cardiovascular death rates, hospitalizations, and urgent heart failure visits in affected patients. It also consistently improves cardiovascular outcomes, functional capacity, and quality of life for those treated.

Detailed Segmentation of the Systemic Amyloidosis Therapeutic Market
The systemic amyloidosis therapeutic market is organized into several key segments:
1) By Product Type: Small Molecule Drugs; Biologic Therapies; Monoclonal Antibodies; Chemotherapy-Based Regimens; Targeted Therapies; Supportive Treatments
2) By Formulation: Tablets; Capsules; Injection
3) By Route of Administration: Oral; Intravenous; Subcutaneous
4) By Distribution Channel: Hospital Pharmacies; Retail Pharmacies; Online Pharmacies
5) By End User: Hospitals; Specialty Clinics; Specialty Pharmacies; Research and Academic Institutes; Other End Users

Further subcategories include:
- Small Molecule Drugs such as Proteasome Inhibitors, Transthyretin Protein Stabilizers, Amyloid Fibril Disruptors, and Immunomodulatory Small Molecules
- Biologic Therapies, including Recombinant Protein Therapies, Gene Silencing Biologic Agents, and Plasma-Derived Products
- Monoclonal Antibodies targeting Amyloid Fibrils, Light Chains, and aimed at targeted clearance
- Chemotherapy-Based Regimens featuring Alkylating Agents, Proteasome Inhibitor Chemotherapy, and Combination Protocols
- Targeted Therapies like Light Chain Suppression, Transthyretin Reduction, and Organ-Specific Treatments
- Supportive Treatments focusing on Cardiac, Renal, Neurological Symptom Management, and Nutritional and Palliative Care

This detailed segmentation provides a comprehensive framework to understand the diverse therapeutic approaches and delivery methods in the systemic amyloidosis market.

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This release was published on openPR.