Amyloidosis is caused by the deposition of insoluble amyloid fibrils formed by the accumulation of misfolded proteins in affected organs. Amyloid immunoglobulin light chain (AL), amyloid A (AA), and amyloid transthyretin (ATTR) amyloidosis are three major subtypes. The only approved regimen to treat adults newly diagnosed with AL amyloidosis is subcutaneous daratumumab in combination with bortezomib, cyclophosphamide, and dexamethasone (D-VCd). Otherwise, treatment consists of the off-label use of other chemotherapy regimens. Treatment of AA amyloidosis relies on the off-label use of IL-6 / IL-1 inhibitors to treat the underlying inflammation. The treatment of ATTR amyloidosis consists of the FDA-approved therapies: Onpattro (patisiran), Tegsedi (inotersen), and Amvuttra (vutrisiran) for the polyneuropathy in these patients and/or Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) for the cardiomyopathy in these patients. Despite these on- and off-label options, there is substantial need for more-effective therapies for all three forms of amyloidosis.
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Content highlights
Geography: United States and EU5.
Primary research: Six country-specific interviews with thought-leading experts in amyloidosis
supported by survey data collected for this study.
Epidemiology: Diagnosed prevalent, incident, and drug-treated cases of amyloidosis by country, segmented by subtype (AL, AA, and ATTR).
Forecast: Drug-level sales and patient share of key amyloidosis therapies through 2032.
Emerging therapies: Phase 3/PR: 5 drugs; Phase 2: 10+ drugs; coverage of select early-phase products
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Niche & Rare Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.
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