Amyloidosis – Landscape & Forecast – Niche & Rare Disease Landscape & Forecast (US/EU5)

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) are three major subtypes of amyloidosis. The only approved regimen for 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. Treatment of ATTR amyloidosis consists of the following FDA-approved therapies: Onpattro (patisiran), Tegsedi (inotersen), Amvuttra (vutrisiran), and Wainua (eplontersen) for the polyneuropathy in these patients and/or Vyndaqel (tafamidis meglumine), Vyndamax (tafamidis) and Attruby (acoramidis) for the cardiomyopathy in these patients. Despite these on- and off-label options, substantial need remains for more-effective therapies for all three forms of the disease.

Questions answered

• What is the size of amyloidosis patient population in the United States and EU5 countries, and how will it change through 2033?
• What is the current treatment landscape and medical practice in amyloidosis, and what are the greatest unmet needs according to experts?
• Which emerging therapies for amyloidosis will launch before 2033, how well will they address the current treatment gaps in this space, and what will be their commercial impact on the amyloidosis market in 2033?

Content highlights

Geography: United States and EU5.

Primary research: Five 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 2033.

Drug treatments: Coverage of key current and emerging therapies

Product description

Niche & Rare Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current and emerging therapies, and drug forecasts supported by detailed primary and secondary research.

Key feature

Niche & Rare Disease Landscape & Forecast features a Drug Pipeline chapter with real-time, global pipeline intelligence content directly from Cortellis. This chapter is updated daily and features interactive figures that can be easily downloaded for detailed analysis or presentations.