https://www.ajmc.com/view/fda-grants-orphan-drug-status-to-regulus-therapeutics-treatment-for-common-type-of-pkd
DA Grants Orphan Drug Status to Regulus Therapeutics’ Treatment for Common Type of PKD
July 29, 2020
Mary Caffrey
Although the name suggests the disease affects only the kidneys, development of fluid-filled cysts can spread to the liver, the pancreas, and other organs. While a healthy kidney is about the size of a fist, a kidney filled with cysts from polycystic kidney disease (PKD) can grow to be about the size of a football weigh up to 30 pounds
Regulus Therapeutics announced today FDA has granted the company orphan drug designation for RGLS4326, an investigational therapy being studied to treat autosomal dominant polycystic kidney disease (ADPKD), the most common type of a group of genetic disorders that lead to end-stage renal disease, morbidity, and early death.