Alnylam’s ONPATTRO™ (patisiran) lipid complex injection is a first-of-its-kind RNA interference (RNAi) therapeutic used to treat the polyneuropathy of hereditary transthyretin-mediated (hATTR) Amyloidosis in adults.
RNAi is a completely new approach to the treatment of these diseases; targeting the faulty protein that is causing the disease rather than treating the symptoms. RNAi therapeutic is an entirely new class of medicines.
In addition to polyneuropathy, hATTR Amyloidosis can lead to other significant disabilities including decreased ambulation with the loss of the ability to walk unaided, a reduced quality of life, and a decline in cardiac functioning.
Patients were randomised in a 2:1 ratio to receive intravenous ONPATTRO (0.3 mg per kg of body weight) or placebo once every 3 weeks for 18 months.
ONPATTRO that received FDA approval was shown to enhance polyneuropathy with reversal of neuropathy impairment in a majority of patients and to improve a composite quality of life measure, reduce autonomic symptoms, and improve activities of daily living.
The FDA approval was made possible due to positive phase 3 results from the APOLLO study which met both on the primary and secondary endpoints.
The study showed that patients treated with Patisiran displayed significant and clinically meaningful improvements in measures of polyneuropathy and quality of life.
The study met on this primary endpoint in that the mNIS+7 score achieved a p-value of p<0>
The endpoint of the APOLLO study was the modified Neuropathy Impairment Score +7 (mNIS+7), which assesses motor strength, reflexes, sensation, nerve conduction and postural blood pressure.
The first FDA approval for an RNAi is helpful to Arbutus Biopharma's LNP technology, which Alnylam had licensed for use.