Inotersen Approved for Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis


The FDA has approved inotersen (TEGSEDI) for the treatment of adults with the polyneuropathy of hereditary transthyretin-mediated amyloidosis.

The US Food and Drug Administration (FDA) has approved inotersen (TEGSEDI) for the treatment of adults with the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR).

“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, chief executive officer at Akcea Therapeutics, in a recent statement.

“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,” she added.

Results from the phase 3 NEURO-TTR study in patients with hATTR amyloidosis with symptoms of polyneuropathy served as the basis for the approval.

Patients treated with inotersen experienced a 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.

Thrombocytopenia and glomerulonephritis are associated risks with inotersen treatment. Consequently, enhanced monitoring is required to detect and manage these risks.

In an exclusive interview with Rare Disease Report®, Morie A. Gertz, MD, oncologist, internist, hematologist, and chair of Internal Medicine, Minnesota, at Mayo Clinic, expressed his optimism surrounding the new drug.

“Now that there’s a rising effective therapy, this disease is a not-to-miss diagnosis. I think there’s going to have to be a major educational initiative for a diagnostic evaluation of this disease to get into the walls of the workflow of neurologists and cardiologists in practice. Now, if you miss it, there really is the potential of harming the patient for failing to provide effective therapy.”

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