ABO-101 Receives Two FDA Designations for Treating PH1

Arbor Biotechnologies announced that the FDA has granted orphan drug designation (ODD) and rare pediatric disease designation (RPDD) to ABO-101 to treat primary hyperoxaluria type 1 (PH1). The rare genetic kidney disease, which often presents in children, is associated with an overproduction of oxalate and can cause kidney stones, kidney damage, and kidney failure.

The ODD and RPDD programs support the development and evaluation of new treatments for serious and life-threatening rare diseases affecting fewer than 200,000 people or children younger than 18 years, respectively. The designations make Arbor eligible for incentives such as tax credits and exemption from certain FDA application fees, as well as potential market exclusivity and a pediatric priority review voucher upon approval.

Arbor will share preclinical data and details on the design of a phase I/II trial of ABO-101 at the 20th Congress for the International Pediatric Nephrology Association in February. The trial will evaluate the drug’s safety, tolerability, pharmacokinetics and pharmacodynamics, and preliminary efficacy in adults and children with PH1.

Source: Press release