The FDA approved inotersen for the treatment of adults with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis, according to the agent’s manufacturer.
Hereditary transthyretin-mediated amyloidosis is a genetic, life-threatening disease that is marked by abnormal buildup of the transthyretin protein in peripheral nerves, the heart and elsewhere in the body.
Inotersen (Tegsedi; Akcea Therapeutics, Ionis Pharmaceuticals) is intended to reduce production of the transthyretin (TTR) protein.
“Tegsedi is the first and only RNA-targeting therapeutic that powerfully reduces the production of TTR protein through a once-weekly subcutaneous injection, offering an effective treatment for people living with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis,” Paula Soteropoulos, CEO of Akcea Therapeutics, said in a company-issued press release. “We believe this profile will make Tegsedi an excellent choice for many patients and that its 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 based this approval on the phase 3, randomized, placebo-controlled NEURO-TTR study of 172 patients with hereditary transthyretin-mediated amyloidosis with symptoms of polyneuropathy.
Researchers observed a 79% mean decrease from baseline in serum transthyretin protein among patients regardless of transthyretin mutation, sex, age or race.
Patients assigned inotersen also experienced significant benefit in measures of neuropathy and quality of life compared with placebo, according to the manufacturer.
“Hereditary transthyretin-mediated amyloidosis is a fatal disease that affects multiple organs and body systems and robs people of so much — the ability to work, the ability to carry out daily tasks — all the things that make them feel in control of their lives,” Morie Gertz, MD, hematologist and chair emeritus of internal medicine at Mayo Clinic, said in the release. “Tegsedi has demonstrated a nearly 80% reduction in transthyretin, which is now a validated approach to treating this disease. The rapid and sustained improvements compared to placebo and reversal in measures of disease seen in a substantial proportion of patients, coupled with the independence offered through self-injection, provide a sense of hope not only to patients, but to their caregivers and families as well.”