Amyloidosis Therapeutics Market to Hit Valuation of US$ 6.37 Billion By 2033 | Astute Analytica

Chicago, May 22, 2025 (GLOBE NEWSWIRE) -- The global amyloidosis therapeutics market was valued at US$ 2.95 billion in 2024 and is expected to reach US$ 6.37 billion by 2033, growing at a CAGR of 9.20% during the forecast period 2025–2033.

The clinical picture of amyloidosis has shifted dramatically since tafamidis became the first therapy to demonstrate a survival benefit in transthyretin (ATTR) cardiomyopathy. By December 2023, U.S. electronic health-record consortium data logged roughly 4,700 newly confirmed ATTR cases—nearly triple the annual figure reported in 2017—underscoring heightened diagnostic vigilance. Alongside this growing patient pool, therapy choice has broadened from a single TTR stabilizer to a spectrum of silencers, stabilizers, fibril disruptors, and transplant adjuncts in the amyloidosis therapeutics market. Vutrisiran’s once-quarterly subcutaneous administration, for instance, cut clinic infusion visits by more than 30,000 nationwide in its first commercial year, alleviating capacity strains at specialty centers. Real-world pharmacovigilance dashboards compiled by FDA Sentinel further show fewer than 45 serious hepatic events per 10,000 patisiran exposures, reinforcing confidence in repeated RNA-based dosing schedules.

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Demand dynamics are equally visible in AL amyloidosis. Autologous stem-cell transplant, long considered the gold standard for fit patients, now competes with monoclonal antibody regimens after the 2024 NCCN inclusion of daratumumab-bortezomib-cytoxan-dexamethasone as a Category 1 option. University of Heidelberg’s amyloid registry reported that 620 German patients initiated antibody-containing induction in 2023, eclipsing transplant volumes for the first time. These shifts reveal a market transitioning from single-pathway dominance to multi-modal management, setting a fertile stage for next-generation candidates that promise earlier intervention, longer dosing intervals, and organ-specific targeting.

Key Findings in Amyloidosis Therapeutics Market

Regulatory Momentum Accelerates Approvals, Offering Clearer Pathways For Innovators Globally

Regulators  in the amyloidosis therapeutics market have moved swiftly to keep pace with the therapeutic surge. Between January 2022 and March 2024, the U.S. FDA, EMA, and Japan’s PMDA collectively granted 17 amyloidosis-related designations—10 orphan, 4 fast-track, 2 breakthrough, and 1 Sakigake—cutting median review timelines by about four months compared with 2018 submissions. The FDA’s June 2023 approval of AstraZeneca-Ionis’s eplontersen for hereditary ATTR polyneuropathy hinged on surrogate neurologic endpoints validated through 1.2 million longitudinal data points from the THAOS registry, a precedent that now guides parallel filings for cardiomyopathic indications.

Policy harmonization is also reducing redundancy. In November 2023, the EMA adopted the FDA’s disease-specific briefing book template, enabling joint scientific advice meetings that two developers—Prothena and BridgeBio—leveraged within 90 days of request. Meanwhile, China’s National Medical Products Administration updated its Rare Disease Catalog to include AL and wild-type ATTR amyloidosis, sparking six domestic IND clearances within 12 months in the amyloidosis therapeutics market. Insurance bodies are responding in lockstep: Australia’s PBAC listed vutrisiran on the Pharmaceutical Benefits Scheme in February 2024 after accepting an adaptive indirect comparison to patisiran, marking the first time a gene-silencing therapy secured reimbursement there without local Phase 3 data. Collectively, these regulatory signals point to an increasingly predictable, data-driven environment that rewards sponsors able to assemble robust natural-history evidence early in development.

Pipeline Diversification Highlights Genomic, Oligonucleotide, And Multispecific Antibody Strategies Emerging

Diversity in the research pipeline is reshaping competitive contours of the amyloidosis therapeutics market. GlobalTrialTracker shows 68 actively recruiting interventional studies for amyloidosis as of April 2024, with mechanisms spanning RNA interference, CRISPR-Cas gene editing, antisense oligonucleotides, and fibril-targeting antibodies. Intellia’s NTLA-2001 remains the most advanced in vivo CRISPR program; interim open-label data released at AHA 2023 revealed a median serum TTR reduction of 92 mg/dL at month 6—sufficient to normalize levels in two-thirds of recipients—while maintaining a manageable infusion-related event count under 12 per cohort.

Multispecific antibodies are gaining momentum in the amyloidosis therapeutics market. CAEL-101, a chimeric fibril-directed IgG1 licensed by AstraZeneca, entered a 540-patient Phase III trial in May 2024 aimed at improving cardiac response in newly diagnosed AL amyloidosis. The study combines the antibody with standard daratumumab-containing regimens, reflecting an industry trend toward backbone-plus-novel-mechanism combinations. On the oligonucleotide front, Alnylam’s ALN-TTRsc04 deployed a GalNAc-enhanced conjugate to extend dosing to biannual intervals; preclinical models demonstrated a 60-day hepatic knockdown plateau with negligible off-target hybridization events, a property now under evaluation in a 120-patient Phase II study. Finally, hot on the heels of positive Phase I read-outs, Prothena’s PRX005—a TTR de-seeding antibody—secured a $150 million option-to-license pact with Bristol Myers Squibb in February 2024. These diversified approaches signal a pivot from symptom mitigation to disease-modifying, and potentially curative, interventions.

Competitive Dynamics Reflect Strategic Collaborations And Acquisitions Across Biopharma Ecosystem

Entrants are choosing partnership over isolation to accelerate timelines and manage cost complexities in the amyloidosis therapeutics market. Alnylam and Regeneron deepened their cardiovascular collaboration in July 2023, earmarking $2.5 billion in potential milestone payments to co-develop a next-generation TTR silencer that leverages Regeneron’s Veloci-Gene platform for hybrid antibody-RNA conjugates. Around the same period, BridgeBio inked a co-promotion deal with Pfizer to expand acoramidis launch capacity across 22 countries pending regulatory nods, granting Pfizer first-right infrastructure in markets where tafamidis already commands formulary familiarity.

M&A activity, while selective, has been strategically timed. Novo Nordisk’s $1.3 billion acquisition of Prothena’s ATTR portfolio in September 2023 delivered a late-stage monoclonal candidate and a preclinical gene-editing program, strengthening Novo’s rare-disease footprint ahead of semaglutide patent cliffs. Venture funding remains robust in the amyloidosis therapeutics market: Series B rounds for start-ups such as Vigil Neuroscience and Tenaya’s amyloid-targeting subsidiary each exceeded $120 million in 2024, channeling capital into novel lipid nanoparticle delivery platforms and cardiac tropic viral vectors. Large-scale CDMO agreements are being finalized sooner; WuXi STA’s 2024 annual report lists nine peptide or oligonucleotide supply contracts tied to amyloidosis programs, up from four in 2021. Collectively, these moves illustrate a competitive field that prizes synergistic capabilities—specialized manufacturing, established commercial channels, and deep disease registry access—over pure asset acquisition, reshaping the power balance among Big Pharma, biotech innovators, and contract organizations.

Diagnostic Advancements Enable Earlier Intervention And Expand Treatable Patient Population

Timely diagnosis remains pivotal, and 2024 saw major gains I nthe amyloidosis therapeutics market. High-sensitivity mass-spectrometry typing, such as Mayo Clinic’s EXENT solution, cut median typing turnaround to 4 days across 15 satellite labs, halving historical delays. At the imaging frontier, Siemens Healthineers’ C-SPECT/CT system obtained FDA 510(k) clearance in October 2023, allowing technetium-99m-PYP scans at community hospitals that previously lacked nuclear cardiology units. This broader availability pushed annual U.S. utilization beyond 56,000 scans—an increase of roughly 18,000 over 2022—capturing patients at NYHA class II rather than class III or IV.

Artificial intelligence is amplifying reach. A Cedars-Sinai-led machine-learning algorithm trained on 1.7 million echocardiograms flagged 14,200 potential wild-type ATTR cases in 2023, prompting confirmatory scintigraphy that verified disease in 2,900 individuals; nearly half commenced TTR-targeted therapy within three months, illustrating AI’s capacity to shorten diagnostic odysseys. Blood-based diagnostics are progressing too in the amyloidosis therapeutics market. C2N Diagnostics’ mis-folded TTR immunoassay, currently in blinded validation involving 10,000 serum samples, aims to deliver a CLIA-certified test by late 2024 capable of distinguishing hereditary from wild-type disease using a five-analyte signature. In aggregate, these tools are redefining the denominator of treatable amyloidosis, feeding more consistent patient flow into therapeutic pipelines and underscoring the market’s reliance on integrated diagnostic-treatment ecosystems.

Market Access Trends Emphasize Value Demonstration And Innovative Payment Contracting

Payer scrutiny has intensified alongside therapy proliferation, pushing sponsors toward creative contracting frameworks in the amyloidosis therapeutics market. In January 2024, Alnylam renewed its outcomes-based agreement with Harvard Pilgrim, stipulating rebates when patisiran-treated patients experience hospitalization rates exceeding predefined ATTR-related thresholds captured through claims data. Since inception, fewer than 190 rebate-trigger events occurred across 4,300 covered lives, illustrating how real-world endpoints can temper budget-impact concerns.

Government insurers are experimenting as well. Italy’s AIFA introduced a risk-share model for vutrisiran in May 2023, linking reimbursement tranches to neurologic improvement scores captured at months 9 and 18. Early readouts showed 740 of 890 enrolled beneficiaries met improvement criteria, unlocking the drug’s final payment installment without dispute. To further anchor cost-effectiveness, manufacturers are bundling diagnostics: BridgeBio’s planned acoramidis launch kit includes no-charge genetic testing via Invitae and one PET-CT voucher, shifting diagnostic outlays away from payers. Meanwhile, ICER’s December 2023 evidence update endorsed tafamidis, vutrisiran, and acoramidis as providing “reasonable long-term value” at U.S. negotiated net prices below $300,000 annually—a benchmark now referenced in at least three state Medicaid renegotiations. Through these multifaceted access mechanisms, the commercial environment is steadily evolving from list-price battles toward agreements that reward verified clinical benefit, ongoing data submission, and shared financial risk.

Regional Outlook Underlines Disparities In Uptake, Infrastructure, And Clinical Capacity

Adoption trajectories vary widely by geography. U.S. specialty pharmacies dispensed approximately 19,000 courses of TTR-targeted therapies in 2023, while combined figures for Germany, France, Italy, Spain, and the United Kingdom hovered near 11,500, reflecting slower cardiology-neurology co-management models in Europe amyloidosis therapeutics market. Japan’s universal coverage boosted tafamidis initiation to 4,200 patients in fiscal-year 2023, aided by 89 certified amyloidosis centers, yet RNA-silencer uptake remained constrained by storage and cold-chain limitations across rural prefectures.

Emerging markets reveal broader gaps. Brazil’s Ministry of Health approved patisiran in August 2023, but public formulary inclusion awaits local pharmacoeconomic analysis slated for Q3 2024. Meanwhile, China’s Hainan Free Trade Port admitted vutrisiran under an early-access scheme, registering 310 treated patients by March 2024; national coverage, however, hinges on Phase III data in an ethnically Han cohort, currently 60 percent enrolled, projected to read out late 2025. Infrastructure disparities in the amyloidosis therapeutics marketalso dictate clinical trial siting: of the 68 open global studies, only 9 list locations in Latin America and Africa combined, perpetuating data deficits in those populations. Nonetheless, targeted investment is emerging—India’s Narayana Health inaugurated a dedicated amyloidosis clinic in December 2023, and Saudi Arabia’s Vision 2030 earmarked funds to expand cardiac amyloid imaging to five tertiary centers. Bridging these regional divides will require coordinated policy, training, and supply-chain enhancements to ensure equitable access.

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Future Outlook Anchored In Personalized Medicine And Real-World Evidence Generation

Looking ahead, the amyloidosis therapeutics market is poised to intersect with personalized medicine trends. Multi-omic profiling, already standard in oncology, is migrating into cardiology clinics; Columbia University’s ATTRomics study, enrolling 2,000 patients, is sequencing whole exomes alongside single-cell transcriptomes to stratify responders versus non-responders across TTR stabilizers, silencers, and gene-editing modalities. These datasets will feed algorithmic treatment pathways integrated into Epic and Cerner systems, guiding drug selection based on mutation type, serum TTR kinetics, and organ involvement scores.

Real-world evidence will further refine value assessment. A 15-year global registry, launched by the Amyloidosis Research Consortium in February 2024, aims to pool de-identified EHR, imaging, and wearable data from at least 25,000 patients. The initiative has already secured data-sharing MOUs with 11 pharma sponsors, ensuring bidirectional feedback loops between clinical outcomes and regulatory commitments. Manufacturing innovation is also on the horizon: Thermo Fisher’s cGMP plasmid facility in Plainville, Massachusetts, scheduled to open late 2024, will triple North American amyloidosis therapeutics market capacity for CRISPR vector production—an essential bottleneck for in vivo gene-editing therapeutics. Coupling these advancements with decentralized trial designs that leverage remote monitoring and home nursing visits could compress recruitment timelines by six months, as projected by recent pilot studies. Taken together, personalization, large-scale real-world analytics, and streamlined manufacturing position the field for quicker iteration cycles and deeper clinical impact, ensuring that the next decade of amyloidosis care will be defined by data-driven precision rather than one-size-fits-all paradigms.

Global Amyloidosis Therapeutics Market Key Players

• Astra Zeneca
• Alnylam Pharmaceuticals, Inc.
• Amgen Inc.
• Bristol-Myers Squibb Company
• F. Hoffmann-La Roche Ltd
• GSK plc.
• Takeda Pharmaceutical Company Limited
• Sanofi, Pfizer Inc.
• Johnson & Johnson Services, Inc.
• Merck KGaA
• Novartis AG
• Ionis Pharmaceuticals
• Other Prominent Players

Key Segmentation:

By Therapy Type

• Chemotherapy
• Stem Cell Transplantation
• Targeted Therapy
• Supportive Care & Surgery
• Others

By Route of Administration

• Oral
• Intravenous
• Subcutaneous

By Indication

• AL Amyloidosis
• ATTR Amyloidosis
• AA Amyloidosis
• Wild-Type ATTR

By End User

• Hospitals & Clinics
• Ambulatory Surgical Centers
• Home Care Settings
• Others

By Distribution Channel

• Hospital Pharmacies
• Retail Pharmacies
• Online

By Region

• North America
• Europe
• Asia Pacific
• Middle East & Africa (MEA)
• South America

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