Akcea Therapeutics, Inc. an affiliate of Ionis Pharmaceuticals, Inc., has announced that Tegsedi (inotersen) has received marketing authorisation approval from the European Commission (EC) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).
hATTR amyloidosis is a progressive, systemic and fatal inherited disease caused by the abnormal formation of the TTR protein and aggregation of TTR amyloid deposits in various tissues and organs throughout the body. The progressive accumulation of TTR amyloid deposits in these tissues and organs leads to sensory, motor and autonomic dysfunction. Ultimately, hATTR amyloidosis results in death within three to fifteen years.
The abnormal formation and aggregation of transthyretin (TTR) protein results in TTR amyloid deposits throughout the body and is the underlying cause of hATTR amyloidosis. Inotersen is designed to block production of the TTR protein. In the NEURO-TTR study, treatment with inotersen produced substantial reductions in the levels of the TTR protein regardless of mutation type or stage of disease.
The European Commission’s approval of inotersen was based on results from the Phase 3 NEURO-TTR study in patients with hATTR amyloidosis, with symptoms of polyneuropathy. Results from that study demonstrated that patients treated with inotersen experienced significant benefit, compared to patients treated with placebo across both co-primary endpoints: the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression.
Paula Soteropoulos, chief executive officer at Akcea Therapeutics said, “With the EC’s decision, inotersen is now the world’s first and only RNA-targeted therapeutic approved for patients with hATTR amyloidosis. This is an important day for the hATTR amyloidosis community as we believe inotersen enables people and their families impacted by this disease to move forward with their lives.”
Teresa Coelho, M.D., neurologist and neurophysiologist at Santo António Hospital, Porto, Portugal said, “hATTR amyloidosis is an inherited, progressive and fatal disease for which treatment options are limited. Inotersen has demonstrated rapid and sustained benefits in improving the course of this disease and preserving quality of life.”