Early access to medicines scheme (EAMS): positive scientific opinion issued for lumasiran in the treatment of primary hyperoxaluria type 1 in all age groups

Positive opinion based on one RCT (n=39 > 6 yrs) which found lumasiran was associated with significant reduction of oxalate in urine of 24 hours by 54% vs. placebo; and single-arm study (n=18 < 6 yrs) which showed consistent results with RCT.

Medicines and Healthcare products Regulatory Agency