Amyloid A (AA) Amyloidosis is a rare disease that is a consequence of chronic inflammatory diseases like rheumatoid arthritis and familial Mediterranean fever and frequently progresses to end-stage renal failure and death. There is no specific treatment approved for use.
New data and a post-hoc analysis of the multinational trial looked at the number of events in the two treatment groups (Fibrillex versus placebo) at each study visit post-baseline. This analysis suggests that treatment with Fibrillex results in an early clinical effect, with fewer patients being classified as ‘worse’ from as early on in the treatment program as month four.
In the study of over 180 AA Amyloidosis patients, the drug appeared to slow disease progression and aid kidney function. Fibrillex reduced the relative risk of doubling their serum creatinine by 54% and reduced by 50% the relative risk of a decrease in their creatinine clearance. The drug also reduced the risk of patients progressing to end-stage renal disease/dialysis or experiencing a decline in their renal functioning.
The data suggest Fibrillex is well tolerated. The most frequent adverse events experienced by the patients in this study were of gastrointestinal origin and infections.