Ionis Pharmaceuticals and affiliate Akcea Therapeutics have secured FDA approval for Tegsedi for treating the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
The approval was based on results from the Phase 3 NEURO-TTR study in patients with hATTR amyloidosis with symptoms of polyneuropathy.
Tegsedi is now approved in the US, Canada and the EU, where it gained marketing approval back in July.
“Tegsedi is the first and only RNA-targeting therapeutic that powerfully reduces the production of TTR protein through a once-weekly subcutaneous injection offering patients an effective treatment for people living with polyneuropathy caused by hATTR amyloidosis,” said Akcea CEO Paula Soteropoulos.
In April, Akcea licensed the worldwide rights to commercialize Tegsedi from Ionis. Based on the US approval of TEGSEDI, Ionis will receive a $50 million milestone payment that may be made in Akcea common stock or cash.