Results from Pediatric Cohort of Phase 1/2 Study of Lumasiran in Patients with Primary Hyperoxaluria Type 1 (PH1) and Insights from a Survey of Physician Experiences with PH1

20 Oct, 2019 Results from Pediatric Cohort of Phase 1/2 Study of Lumasiran in Patients with Primary Hyperoxaluria Type 1 (PH1) and Insights from a Survey of Physician Experiences with PH1

We presented results on the pediatric cohort of patients (N=16; age range 6-17) from the Phase 1/2 study of lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of primary hyperoxaluria type 1 (PH1), at the International Pediatric Nephrology Association (IPNA) 2019 Annual Meeting, held October 17-21 in Venice, Italy.

Frishberg et al. – Phase 1/2 Study of Lumasiran, Investigational RNAi Therapeutic, in Patients with Primary Hyperoxaluria Type 1

Danese et al. – Understanding the Burden of Primary Hyperoxaluria Type 1 (PH1): A Survey of Physician Experiences with PH1

Pediatric patients receiving lumasiran experienced clinically significant and sustained reductions in urinary oxalate to normal or near normal levels, with a safety and tolerability profile consistent with the overall patient population (N=20).

In addition, Alnylam and collaborators reported on results from a survey of physician experiences with PH1 providing insight on the factors that contribute to the burden of disease throughout the patient journey, including recurrent kidney stones, frequent surgical intervention and hospitalizations and, if untreated, progression to end-stage renal disease, requiring intensive dialysis and eventual organ transplantation.