Onpattro

Onpattro (patisiran) is a medication indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy. The regulatory review process included an Orphan Medicinal Product Designation on 15 April 2011, followed by a Marketing Authorisation Application Submission Date on 18 December 2017, and a CHMP Positive Opinion Date on 26 July 2018.

In a Phase 3 study, the primary efficacy endpoint was the change from baseline in the modified Neuropathy Impairment Score (mNIS+7) at 18 months. Patisiran-LNP demonstrated a least squares mean difference of -30.63 points compared to placebo. The proportion of patients with a less than 0-point increase in mNIS+7 score was 56.1% in the patisiran group, compared to 3.9% in the placebo group, resulting in a difference in proportions of 52.2. The odds ratio for this outcome was 39.9.

Regarding safety, arthralgia was reported in 7.4% of patients in the patisiran-LNP group. The rate of death per 100 patient-years was 6.2 in one group and 3.2 in another. Cardiac hospitalization and/or death occurred at a rate of 10.1 per 100 patient-years. There were 6 and 7 deaths reported in different groups, respectively. Patients with any hospitalization and/or death numbered 31 and 51 in different groups.