the US Food and Drug Administration (FDA) approved Tegsedi (inotersen), an antisense oligonucleotide (ASO) that inhibits the production of the transthyretin (TTR) protein (amyloid), for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) in adults. This is the second FDA approved drug for hATTR. Onpattro (patisiran), was the first treatment approved for this indication on August 10, 2018.
hATTR is a rare, debilitating, and often fatal genetic disease characterized by the buildup of abnormal amyloid protein in peripheral nerves, the heart, and other organs, interfering with their normal functioning. The clinical signs and symptoms of amyloid deposition in the peripheral nervous system can include effects on sensation (pain and temperature), autonomic function (blood pressure changes and bowel and digestive problems), and muscle strength (weakness and immobility in the arms, legs, hands, and feet).
Tegsedi is contraindicated in patients with a history of acute glomerulonephritis (inflammation of filters in the kidney called glomeruli) caused by Tegsedi, patients with a history of a hypersensitivity reaction to Tegsedi, and patients with a platelet count less than 100 x 109 /L. Tegsedi’s label contains a Boxed Warning about thrombocytopenia (low blood platelet count) which may be life threatening, and glomerulonephritis, which may require immunosuppressive treatment and may result in dialysis-dependent renal failure.
Laboratory monitoring for both conditions is required before, during, and after treatment discontinuation. Because of the risks of serious bleeding due to severe thrombocytopenia and glomerulonephritis, Tegsedi is available only through a restricted distribution program under a Risk Evaluation and Mitigation Strategy (REMS) called the Tegsedi REMS Program.
(Source: The U.S. Food & Drug Administration)