Rare Daily Staff

The European Commission approved Akcea Therapeutics’ and Ionis Pharmaceuticals’ Tegsedi for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis, a progressive, systemic, and fatal inherited disease.

In April, Akcea licensed the worldwide rights to commercialize Tegsedi from Ionis. Based on the European Commission authorization of Tegsedi, 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 percent to Ionis and 40 percent to Akcea.

Hereditary transthyretin amyloidosis is caused by the abnormal formation of the TTR protein and aggregation of TTR amyloid deposits in various tissues and organs throughout the body, including in peripheral nerves, heart, intestinal tract, eyes, kidneys, central nervous system, thyroid and bone marrow. The progressive accumulation of TTR amyloid deposits in tissues and organs leads to sensory, motor, and autonomic dysfunction. Patients with hATTR amyloidosis often experience nerve degeneration and damage to the heart. Ultimately, hATTR amyloidosis results in death within three to fifteen years of symptom onset.

Therapeutic options for the treatment of patients with hATTR amyloidosis are limited and there are currently no disease-modifying drugs approved for the disease. There are an estimated 50,000 patients with hATTR amyloidosis worldwide.

Tegsedi is an antisense oligonucleotide inhibitor of human transthyretin production. It is designed to block production of the TTR protein.

The approval is based on data from the NEURO-TTR study which was a late-stage, randomized, double-blind, placebo-controlled, international study in 172 patients with hATTR amyloidosis with symptoms of polyneuropathy. The 15-month study measured the effects of Tegsedi on neurological function and on quality-of-life by measuring the change from baseline in the modified Neuropathy Impairment Score +7 and in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy total score. Tegsedi provided significant benefit on both co-primary endpoints.

In the European Union, Tegsedi is approved for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis. The European Commission marketing approval follows the positive opinion recommending approval provided by the Committee for Medicinal Products for Human Use of the European Medicines Agency.

It is under regulatory review in the United States and Canada. The U.S. Food and Drug Administration is expected to act on the company’s application for market approval by October 6.

Tegsedi is associated with risk of thrombocytopenia (low platelet count) and glomerulonephritis (acute inflammation of the kidney). Enhanced monitoring is required to support early detection and management of these identified risks.

“With the EC’s decision, Tegsedi is now the world’s first and only RNA-targeted therapeutic approved for patients with hATTR amyloidosis,” said Paula Soteropoulos, CEO of Akcea. “With subcutaneous delivery, Tegsedi puts treatment in the patients’ hands while bringing the significant benefits shown in the NEURO-TTR study in both measures of neuropathy and quality of life for people living with this serious and fatal disease.”

July 17, 2018
Paula Soteropoulos, CEO of Akcea