Remove Clinical Development Remove Gene Silencing Remove Genetic Disease Remove Genotype
article thumbnail

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

The Pharma Data

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. The safety and efficacy of Oxlumo are also being evaluated in the ongoing ILLUMINATE-C Phase 3 clinical trial in patients of all ages with advanced PH1, including patients on dialysis. Oxlumo should be administered by a healthcare professional.