Therapeutics indicated for the treatment of transthyretin-associated amyloidosis in adult patients with symptomatic stage 1 polyneuropathy to delay peripheral neurologic compromise.
Treatment should be initiated and maintained under the supervision of a physician familiar with the treatment of patients with transthyretin-associated amyloid polyneuropathy.
The drug should be added to standard treatment for the treatment of patients with transthyretin-associated familial amyloid polyneuropathy (PAF-TTR). Physicians should monitor patients and continue to evaluate the need for other therapies, including the need for hepatic transplantation, as part of this standard treatment. As data on the use of the medicinal product after hepatic transplantation are not available, it should be discontinued in patients undergoing liver transplantation.
Source: EMA