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With Lumasiran Pediatric Data, Alnylam Hopes For Approvals In All PH1 Patients

 
• By Joseph Haas

Alnylam shows that lumasiran offers similar safety and efficacy in primary hyperoxaluria type 1 patients who are six or younger as it does in older patients. US and EU regulatory decisions loom.

Kidney healthcare, doing medical research
Alnylam waits for US and EU regulatory decisions in rare kidney disorder

Alnylam Pharmaceuticals Inc. detailed its pediatric data for lumasiran in primary hyperoxaluria type 1 (PH1) on 22 October, showing the RNA-interference therapy works roughly as well and with similar safety as in older patients. Following a top-line readout of the ILLUMINATE-B study on 30 September, Alnylam now looks ahead to approval decisions for lumasiran in the US and EU with hopes that the therapy might be approved for patients of all ages.

ILLUMINATE-B studied lumasiran using weight-based, quarterly dosing in 18 patients under the age of six years, with the youngest initiating therapy at three months of age. On the primary endpoint, the study showed a 72% mean reduction in urinary oxalate at six months from baseline. In June, Alnylam reported that the drug yielded a mean 65

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