FDA Grants ABO-101 Orphan Drug, Rare Pediatric Disease Designations for PH1
Primary hyperoxaluria type 1 (PH1) is a rare genetic condition that affects the kidney, bladder, or urinary tract, and indicates high levels of oxalates in the urine.
The FDA granted ABO-101 orphan drug and rare pediatric disease designations for the treatment of primary hyperoxaluria type 1 (PH1). This designation follows the clearance of the investigational new drug application for the gene editing therapeutic in December 2024.1
This designation follows the clearance of the investigational new drug application for the gene editing therapeutic in December 2024. | Image Credit: James Thew | stock.adobe.com
“ABO-101 receiving rare pediatric disease and orphan drug designations from the FDA for the potential treatment of PH1 underscores the urgent need for novel treatment options,” Dan Ory, MD, chief medical officer of Arbor Biotechnologies, said in a news release.1 “As Arbor advances ABO-101 into the clinic with the initiation of the redePHinephase 1/2 clinical study, these designations reinforce the potential of ABO-101 to deliver lasting disease modification as a first-in-class gene editing therapy for PH1.”
ABO-101 is a novel, investigative gene editing medication that is intended to be a one-time therapy. The medication is directed at the loss of function of the HAO1 gene in the liver, which reduces PH1-associated oxalate production.1
Arbor Biotechnologies will present preclinical data that supports the drug as well as showcasing safety, tolerability, pharmacokinetic/pharmacodynamic, and preliminary efficacy for both adults and pediatric patients. This data will be presented at the 20th Congress for the International Pediatric Nephrology Association from February 19 to February 23, 2025, in Cape Town, South Africa.1
In May 2024, Arbor presented preclinical data on the potential of ABO-101 at the 2024 American Society of Gene and Cell Therapy 27th Annual Meeting in Baltimore, Maryland. The therapeutic potential from in vivo data showed that the drug has highly specific targeting of HAO1 in the level and preservation of genomic integrity. Further, it resulted in a corresponding therapeutically relevant reduction in urinary oxalate and was well tolerated in nonhuman primates at multiple doses. The data also confirmed the efficacy and pharmacology in the drug.2
PH1 is a rare and serious condition that affects the kidney, bladder, or urinary tract, and indicates high levels of oxalates in the urine, according to the National Kidney Foundation. Oxalate is found in many foods but is also considered a waste product produced by the body naturally. Kidneys filter out oxalate via the blood and urine, with people who have PH1 experiencing high levels of oxalate, which increases the risk of calcium oxalate stone and, in serious cases or being left untreated can cause kidney failure.3
Symptoms of PH1 include kidney stones, sharp pain in the back, side, or grain areas, blood in urine, frequently urinary tract infection, urinary incontinence, painful urination, needing to urinate often, or not being able to urinate or urinate in small amounts. Symptoms can develop at any time, though most patients develop symptoms as a child, as early as less than 1 year, into young adulthood. PH1 is a genetic condition, therefore, it is passed down from parent to child.3
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