IONIS-TTRRx filed with EMA for hereditary TTR amyloidosis.- Ionis Pharma.
Ionis Pharmaceuticals has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for IONIS-TTRRx (inotersen), based on the phase III NEURO-TTR study in patients with hereditary TTR amyloidosis (hATTR). The MAA for inotersen will be reviewed under the EMA's Accelerated Assessment program, which is intended to expedite access to drugs that the EMA considers to be of major therapeutic interest. Next week, the company also plans to submit the NDA to the FDA.
The application is based on a completed Phase III study, NEURO-TTR, in patients with polyneuropathy due to hereditary TTR amyloidosis (hATTR) which was reported 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. Inotersen-treated patients benefitted significantly in the quality of life primary endpoint compared to placebo, with a difference in magnitude of 11.68 points in the Norfolk QoL-DN score at 15 months of treatment (mean change from baseline of 0.99 vs. 12.67, p=0.0006).
The overall mortality rate in the NEURO-TTR study was 2.9% and was lower than mortality rates reported in other studies in hATTR patients. There was a total of five deaths in the study, five (4.7%) in the inotersen arm and zero in the placebo arm. Four deaths in the inotersen arm were associated with disease progression and considered unrelated to treatment. As previously reported, there was one fatal intracranial hemorrhage in conjunction with serious thrombocytopenia. No serious thrombocytopenia was observed following implementation of more frequent monitoring.
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