CHMP recommends approval of Tegsedi for hereditary transthyretin amyloidosis.- Ionis USA.
The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Tegsedi (inotersen), from Ionis USA, intended for the treatment of hereditary transthyretin amyloidosis. Tegsedi, which was designated as an orphan medicinal product on 26 March 2014, was reviewed under EMA’s accelerated assessment programme. Tegsedi will be available as a 284-mg solution for injection.
Tegsedi has shown clinically relevant effects on both the neurological components of the disease and on quality of life. The most important side effects are injection site reactions, thrombocytopenia and glomerulonephritis. The full indication is: "Treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR)”.
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