he US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) and rare paediatric disease designation (RPDD) to Arbor Biotechnologies’ gene editing therapeutic, ABO-101, to treat primary hyperoxaluria type 1 (PH1).
The orphan drug and rare paediatric disease designations will offer the company incentives such as tax credits, exemption from certain regulatory application fees, potential market exclusivity and eligibility for a paediatric priority review voucher upon the approval of the therapy.
The development comes after the US regulator granted approval for the investigational new drug (IND) application for the therapeutic in December 2024.
The company anticipates commencing the Phase I/II redePHine study in the first half of 2025.
The trial will assess the tolerability, pharmacokinetics, safety, preliminary efficacy and pharmacodynamics of the therapeutic in adult and paediatric patients.
Arbor Biotechnologies chief medical officer Dan Ory stated: “ABO-101 receiving rare paediatric disease and orphan drug designations from the FDA for the potential treatment of PH1 underscores the urgent need for novel treatment options.
“As Arbor advances ABO-101 into the clinic with the initiation of the redePHine Phase I/II 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 one-time liver-directed gene editing treatment tailored to cause the permanent function loss of hydroxyacid oxidase 1(HAO1) gene in the liver, thus minimising the production of oxalate associated with PH1.
This therapy employs an Acuitas Therapeutics-licensed lipid nanoparticle (LNP), encapsulating mRNA that expresses a novel Type V CRISPR Cas12i2 nuclease alongside an optimised guide RNA targeting the HAO1 gene of the human.
Often manifesting in children, PH1 leads to excess oxalate production, resulting in stone formation in the kidney and potentially leading to failure.
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