2018/01/25

FDA grants Orphan Drug Designation to inclisiran for the treatment of homozygous familial hypercholesterolemia (HoFH)
25 Jan 2018


INCLISIRAN’S FIRST PIVOTAL TRIAL ACHIEVES TARGET ENROLLMENT AHEAD OF SCHEDULE
– ORION-11, a Phase III confirmatory clinical trial randomized more than 1,500 patients in 11 weeks –
FDA grants Orphan Drug Designation to inclisiran for the treatment of homozygous familial hypercholesterolemia (HoFH)
– Following encouraging Phase II data, a Phase III clinical trial of inclisiran in HoFH set to begin in 2018 –
The Medicines Company (NASDAQ: MDCO) today announced that the target of 1,500 patients randomized in the ORION-11 trial has been exceeded in 11 weeks – considerably beating its goal of 28 weeks. ORION-11 is a double-blind, randomized Phase III trial designed to confirm the effectiveness and safety of inclisiran, an investigational agent which is potentially a first-in-class lipid-lowering drug, to reduce LDL-cholesterol (LDL-C) in patients with atherosclerotic cardiovascular disease (ASCVD) or cardiovascular risk-equivalents. In the trial, patients are randomized to inclisiran 300 mg or placebo given subcutaneously on days 1, 90, 270 and 450 – then followed to the end of the study at 540 days (18 months). ORION-11 is one of three similar trials designed to confirm inclisiran safety and effectiveness and potentially provide key data required for NDA and MAA submission in the United States and Europe. The other two trials (ORION-9 in heterozygous familial hypercholesterolemia and ORION-10 in ASCVD) are actively enrolling – also ahead of schedule – and are expected to complete enrollment during the first half of 2018. A fourth pivotal Phase III study in patients with HoFH is expected to enroll patients beginning later in 2018 and, following 6 months of treatment, to provide data for HoFH as part of a comprehensive LDL-C lowering program in a planned NDA and MAA submission.