12 January 2018 - - Carlsbad, California-based RNA-targeted therapeutics developer Ionis Pharmaceuticals, Inc.'s (NASDAQ: IONS) new drug application (NDA) for inotersen has been accepted for Priority Review by the US Food and Drug Administration, the company said.
The FDA has set a Prescription Drug User Fee Act (PDUFA) date of July 6, 2018.
Inotersen is an investigational antisense drug designed to reduce the production of transthyretin, or TTR, for the treatment of patients with hereditary TTR amyloidosis (hATTR), a severe, rare, and fatal disease.
Results of the NEURO-TTR Phase 3 study of Inotersen in patients with polyneuropathy due to hereditary TTR amyloidosis (hATTR) completed in May 2017 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 also benefited significantly in the co-primary endpoint of disease control, mNIS+7, with 47% of patients experiencing improved or stable scores compared to baseline and a mean difference in magnitude of 19.73 -points, compared to placebo-treated patients, at 15 months of treatment, (p < 0.001).
Two key safety issues were identified during the study: thrombocytopenia and safety signals related to renal function.
Enhanced monitoring was implemented during the study to support early detection and management of these issues. Serious platelet and renal events were infrequent and easily managed with routine monitoring, which has proven effective since implementation.
Ionis is focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases. Using its proprietary antisense technology, the company has created a large pipeline of first-in-class or best-in-class drugs, with over three dozen drugs in development.