FDA Approves Akcea and Ionis’ Drug for hATTR Amyloidosis

Rare Daily Staff

The U.S. Food and Drug Administration approved Akcea Therapeutics and Ionis Pharmaceuticals’ Tegsedi for the treatment of adult polyneuropathy of hereditary transthyretin-mediated amyloidosis, a progressive, systemic, and fatal inherited disease.

Hereditary transthyretin amyloidosis (hATTR) 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 15 years of symptom onset.

Tegsedi is an antisense oligonucleotide inhibitor of human transthyretin production. It is designed to block production of the TTR protein. The drug is now approved in the United States, European Union, and Canada. The FDA approved the drug with a black box warning because it can potentially cause a dangerous drop in users platelet counts and result in serious bleeding.

In April, Akcea licensed the worldwide rights to commercialize Tegsedi from Ionis. Based on the U.S. approval of Tegsedi, Ionis will receive a $50 million milestone payment that may be made in Akcea common stock or cash. Commercial profits and losses from Tegsedi will be split 60 percent to Ionis and 40 percent to Akcea.

“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 Paula Soteropoulos, CEO of Akcea Therapeutics. “We believe this profile will make Tegsedi an excellent choice for many patients and that it’s self-administration gives the flexibility to treat at a time that works for them which could change the way this progressive and debilitating disease is treated and managed.”

The FDA’s approval of Tegsedi was based on results from the phase 3 NEURO-TTR study in patients with hATTR amyloidosis with symptoms of polyneuropathy. The study showed that patients treated with Tegsedi experienced significant benefit compared to patients treated with placebo across both co-primary endpoints of standardized measures of neuropathy and the progression of neuropathic disease.

Because of safety concerns, enhanced monitoring is required to support early detection and management of identified risks associated with use of the drug.

To assist hATTR amyloidosis patients in gaining access to Tegsedi, Akcea has created a patient support program. This program offers free, private, and personalized support to patients and their caregivers and families across the United States.

October 8, 2018
Photo: Paula Soteropoulos, CEO of Akcea Therapeutics