The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the granting of a marketing authorisation for tafamidis (Vyndaqel) intended for the treatment of transthyretin amyloidosis in adult patients with symptomatic polyneuropathy, a severe, progressive orphan disease. Although the applicant was not able to provide comprehensive evidence on the efficacy and safety of this medicine, the positive opinion was granted under exceptional circumstances, due to the rarity of the disease.

Tafamidis is the first oral pharmacological treatment recommended for this rare disease.