Tegsedi approved in Europe for rare hereditary disease

As was largely expected, Tegsedi (inotersen) has received marketing authorization approval from the European Commission for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).

This follows the positive opinion recommending approval provided by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency in June.

In April, Akcea Therapeutics (Nasdaq: AKCA) licensed the worldwide rights to commercialize Tegsedi from Ionis Pharmaceuticals (Nasdaq: IONS). Based on the EC authorization of the drug, Ionis will receive a $40 million milestone payment from Akcea payable in shares of Akcea common stock. Commercial profits and losses from Tegsedi will be split 60% to Ionis and 40% to Akcea. Akcea shares rose nearly 5% Wednesday.