Shots:

• The P-III ILLUMINATE-B study will assess Lumasiran in 8 patients with primary hyperoxaluria type 1 (PH1) aged < 6yrs.- associated 1EPs as reduction of urinary oxalate in 6mos. & 2EPs as evaluating glomerular filtration rate (GFR)- safety- tolerability- with its expected initial results in mid-2020
• The company also reports the result of P-II OLE study result assessing lumasiran (1.0 & 3.0 mg/kg monthly- and 3.0 mg/kg quarterly) in patients and resulted in 72% reduction in urinary oxalate excretion- mean reduction relative to baseline 69%; 77% reduction in urinary 24-hour oxalate : creatinine ratio
• Lumasiran (formerly ALN-GO1- SC) is an RNAi therapeutics targeting glycolate oxidase (GO) and has received FDA’s BT & ODD- EU’s ODD & EMA’s PRIME Designation

Ref: Alnylam | Image: FDA Headlines