Research & Development
Akcea and Ionis announce EU approval for hATTR amyloidosis treatment
11 July 2018 -

Akcea Therapeutics Inc (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals Inc (NASDAQ: IONS), has received marketing authorisation approval for TEGSEDI (inotersen) in the European Union, the company said on Wednesday.

The European Commission (EC) approved the drug for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin (hATTR) amyloidosis.

An inherited, progressive disease, hATTR amyloidosis is caused by a mutation in the transthyretin (TTR) gene that leads to the abnormal formation and aggregation of TTR protein, resulting in TTR amyloid deposits throughout the body.

TEGSEDI is designed to block production of the TTR protein. In the Phase 3 NEURO-TTR study, treatment with TEGSEDI produced substantial reductions in the levels of the TTR protein regardless of mutation type or stage of disease.

The EC decision means that TEGSEDI is now the world's first and only RNA-targeted therapeutic approved for patients with hATTR amyloidosis, according to Akcea CEO Paula Soteropoulos.

"We are ready to launch TEGSEDI along with our patient and physician support services across Europe," Soteropoulos added.