The Food and Drug Administration (FDA) has approved Tegsedi (inotersen; Akcea Therapeutics and Ionsis) for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR), a rare, progressive and debilitating genetic disease caused by a mutation in the transthyretin (TTR) protein.

Tegsedi is an antisense oligonucleotide that causes degradation of mutant TTR mRNA through binding to the TTR mRNA, which results in a reduction of serum TTR protein and TTR protein deposits in tissues. The approval was based on data from the Phase 3 NEURO-TTR study (N=173) which randomized patients with polyneuropathy caused by hATTR amyloidosis to Tegsedi or placebo.

Results showed that treatment with Tegsedi was associated with significant clinical benefit compared with placebo across both co-primary endpoints: the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and modified Neuropathy Impairment Scale +7 (mNIS+7), a measure of neuropathic disease progression.

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“Tegsedi has demonstrated a nearly 80% reduction in TTR, which is now a validated approach to treating this disease,” said Morie Gertz, MD, hematologist and Chair Emeritus of Internal Medicine at Mayo Clinic. “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.”

With regard to safety, Tegsedi carries a Boxed Warning regarding the risk of thrombocytopenia and glomerulonephritis. Other side effects associated with therapy include injection site reactions, nausea, headache, fatigue, and fever. Tegsedi is available only through a restricted distribution program called the Tegsedi REMS program.

Tegsedi is supplied in a single-dose, prefilled syringe that is filled to deliver 1.5mL of solution containing 284mg of inotersen and is intended for subcutaneous use only.

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