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ABO-101 Receives FDA Orphan Drug and Rare Pediatric Designations for Primary Hyperoxaluria Type 1
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Key Takeaways
- ABO-101 has received FDA orphan drug and rare pediatric disease designations for treating primary hyperoxaluria type 1 (PH1).
- PH1 is a genetic condition causing excessive oxalate production, leading to kidney stones and systemic oxalosis.
ABO-101 is supported by preclinical data that demonstrated significant reductions in urinary oxalate in PH1 disease models.
The FDA has granted orphan drug designation (ODD) and rare pediatric disease designation (RPDD) to ABO-101 (Arbor Biotechnologies), a gene editing therapy indicated to treat primary hyperoxaluria type 1 (PH1). Preclinical data outlining the safety, tolerability, and preliminary efficacy of ABO-101 among adult and pediatric patients with PH1 will be presented at the 20th Congress for the International Pediatric Nephrology Association (IPNA) later this month.1
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“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. “As Arbor advances ABO-101 into the clinic with the initiation of the redePHine Phase 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.”1
According to the National Kidney Foundation, PH1 is a rare and genetic condition that commonly impacts the kidneys, bladder, or urinary tract. The condition is caused by enzyme deficiencies in the liver, which can lead to the overproduction and buildup of oxalate. Individuals with PH1 have significantly high levels of oxalate in their urine, placing them at increased risk of kidney stones, early-stage kidney disease, and systemic oxalosis.1,2
Although symptoms of PH1 can vary, the most common signs include frequent kidney stones; sharp pain in the back, side, or groin area; blood in the urine; frequent urinary tract infections; painful urination; urinary incontinence; and frequent urge to pee. Symptoms can start to develop in infants less than 1 year old, along with children and young adults.2
Treatment options for individuals with PH1 can vary depending on the severity of the case. Currently, lumasiran (Oxlumo; Alnylam Pharmaceuticals, Inc.) and nedosiran (Rivfloza; Novo Nordisk) are the only FDA-approved injections indicated for PH1. However, other nonprescription treatments include drinking plenty of fluids, taking vitamin B6, using citrate or bicarbonate salts, avoiding high amounts of vitamin C, and lowering the amount of oxalate consumed in foods.2
ABO-101 could provide a durable reduction in oxalate production because it is indicated to diminish HAO1 gene expression in the liver. The study authors noted that the gene editing therapy includes a lipid nanoparticle that encapsulates messenger RNA expression, a novel Type V CRISPR Cas12i2 nuclease, and an optimized guide RNA, targeting the human HAO1 gene.1
The FDA ODD and RPDD designations follow the FDA clearance of the investigational new drug (IND) application for ABO-101, granted in December 2024. The study authors noted that ABO-101 was supported by preclinical data that demonstrated specific and durable in vivo editing of HAO1 and significant reductions in urinary oxalate in PH1 disease models.3
“The initiation of this study marks an important milestone both for patients with PH1 and in the growth of our company as we begin advancing our pipeline of first-in-class programs into the clinic,” Devyn Smith, CEO of Arbor, said in a news release. “There is a tremendous need for innovative approaches to treat genetic conditions—from ultra-rare to the most common genetic diseases.3
