TEGSEDI(inotersen)

TEGSEDI (inotersen) is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. It received Orphan Medicinal Product Designation on 23 March 2014 and was approved on 10/25/2019.

In clinical trials, TEGSEDI demonstrated a treatment difference (LS Mean) in mNIS+7 of -19.7 compared to baseline, with a p-value of <0.001. The mean percent decrease from baseline in serum TTR ranged from 68 to 74%, and the median percent decrease was 75 to 79%. The drug also showed a mean reduction in serum retinol binding of 71% and a mean reduction in serum vitamin A of 63%.

Safety findings included a 14% incidence of platelet count below 75 x 10^9/L, compared to 0% in placebo. Severe thrombocytopenia (platelet count below 25 x 10^9/L) occurred in 3% of patients. Glomerulonephritis was observed in 3% of patients, while 15% experienced a urine protein to creatinine ratio greater than 5 times the upper limit of normal, compared to 8% in placebo. An increase from baseline in serum creatinine greater than 0.5 mg/dL was seen in 11% of patients.