Amyloidosis | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2016
Amyloidosis refers to the disruption and damage to organs and tissues due to deposits of abnormal, insoluble amyloid fibrils, which are formed by the accumulation of misfolded proteins. Amyloid immunoglobulin light chain (AL), amyloid A (AA), and amyloid transthyretin (ATTR) are three major subtypes of amyloidosis, termed after the corresponding protein precursor. Treatment options for multiple myeloma are used off-label for AL amyloidosis. In 2011, the EMA approved the TTR stabilizer tafamidas—Pfizer’s Vyndaquel—for ATTR-familial amyloid polyneuropathy; it is the only drug ever to gain approval for any subtype of amyloidosis. Thanks to the growing knowledge of the pathogenesis of amyloidosis, several agents with novel mechanisms of action are in late-stage development and likely to reach the market in the near term.
