Kaerus Bioscience secures FDA approval for rare genetic disorder treatment
Ella Day | May 15, 2025 | News story | Research and Development | FDA, Fragile X syndrome, Kaerus Bioscience, Orphan Drug Designation (ODD), Rare Diseases, Rare Pediatric Drug Designations (RPDD), genetic syndromes
Kaerus Bioscience announces that its lead candidate KER-0193 has been granted both Orphan Drug Designation (ODD) and Rare Pediatric Drug Designations (RPDD) for the treatment of Fragile X syndrome (FXS) by the US Food and Drugs Administration (FDA). Created by Medicxi, the global biopharma is focused on the development of therapeutics for rare genetic syndromes of neurodevelopment. This is a significant milestone in Kaerus’ aim to deliver the first effective treatment for the disorder.
The approval follows the successful outcome of a phase 1 trial of KER-0193 which investigated its effect on brain activity using electroencephalography. It demonstrated that the drug is safe and tolerable, affirming Kaerus’ preclinical animal studies and data.
“These important FDA designations are more excellent news for Kaerus coming after the impressive phase 1 results showing KER-0193,” commented Paul Sekhri, chairman of Kaerus.
KER-0193 seeks to address hyperexcitability of brain function commonly associated with FXS by targeting calcium activated potassium channels which are understood to have reduced function in individuals with FXS.
FXS is an inherited condition and the most common cause of inherited autism and intellectual disability. Those with FXS can experience a range of behavioural, sensory and cognitive challenges, and currently there are no approved treatments.
Robert Ring, CEO of Kaerus, said: “The FDA’s granting of ODD and RPDD for KER-0193 is an important step towards our objective of delivering an effective treatment for people with FXS.”
The company is finalising preparations for a phase 2 study in FXS patients.
Ella Day
15/5/25
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