Remove Genetic Disease Remove Genotype Remove HR Remove Medicine
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. On November 19, the European Commission granted marketing authorization for Oxlumo for the treatment of PH1 in all age groups, following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP). 0.514 mmol/24 hr/1.73