Investigational drug filed for the treatment of hereditary TTR amyloidosis (hATTR)

Ionis Pharmaceuticals announced that its New Drug Application (NDA) for IONIS-TTRRx (inotersen) has been accepted for Priority Review by the FDA. Inotersen is an investigational drug for the treatment of patients with hereditary TTR amyloidosis (hATTR). The FDA has set a Prescription Drug User Fee Act (PDUFA) date of July 6, 2018.

Inotersen completed a Phase III study, NEURO-TTR, in patients with polyneuropathy due to hereditary TTR amyloidosis (hATTR) in May 2017. Results from the study demonstrated benefit compared to placebo across both primary endpoints of the study: the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and the modified Neuropathy Impairment Score +7 (mNIS+7) at both eight and 15 months of treatment. In addition, consistent and significant benefit was observed in both the Norfolk-QoL-DN and mNIS+7, independent of disease stage, types of mutation, previous treatment with TTR protein stabilizers or presence of cardiomyopathy at the beginning of the study.

Inotersen-treated patients benefited significantly in the quality of life primary endpoint with 50% of patients experiencing improved scores compared to baseline and a mean difference in magnitude of 11.68 points, compared to placebo-treated patients, at 15 months of treatment (mean change from baseline of 0.99 vs. 12.67). The overall mortality rate in the NEURO-TTR study was 2.9% and was lower than overall mortality rates reported in other studies in hATTR patients.