Kaerus secures FDA designations for Fragile X syndrome treatment

Fragile X treatment KER-0193 gets FDA orphan, rare disease tags

KER-0193, an oral therapy being developed as a fragile X syndrome treatment, has been awarded orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). These designations — which follow news of positive Phase 1 trial data from a test in healthy volunteers and plans for a Phase 2 trial in fragile X patients — are “an important step towards our objective of delivering an effective treatment for people with…

Kaerus Bioscience secures FDA approval for rare genetic disorder treatment - Pharmafile

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 […] The post Kaerus Bioscience secures FDA approval for rare genetic disorder treatme…

Kaerus secures FDA designations for Fragile X syndrome treatment

Kaerus Bioscience has received US Food and Drug Administration’s (FDA) designations for KER-0193 to treat Fragile X syndrome (FXS).

Kaerus Bioscience's lead candidate KER-0193 granted Orphan Drug Designation and Rare Pediatric Drug Designation by U.S. FDA for treatment of Fragile X syndrome (FXS) - PressReach

FDA’s two designations demonstrate KER-0193’s potential as a treatment for FXS Follows the successful completion of Phase 1 clinical trial Fragile X syndrome is the most common cause of inherited autism LONDON, May 14, 2025 /PRNewswire/ — Kaerus Bioscience (“Kaerus” or “the Company”), a clinical stage biopharmaceutical company created by Medicxi for the development of therapeutics for rare genetic syndromes of neurodevelopment, today announces t…

Fragile X Syndrome Workshop Invitation

We are delighted to let you know about a forthcoming International Workshop on Fragile X Syndrome on June 20th 2025 in Padua, organised through Montore Sanita. By kind invitation of Dr Elisa Di Giorgio, newly appointed member of our Board of Scientific and Clinical Advisors, it is open to anyone interested both in person and live on Zoom or Facebook. Please see the attached letter and programme. To register go to https://www.motoresanita.it/even…

Kaerus Bioscience secures orphan drug designation for fragile x syndrome treatment

FDA recognises KER-0193 as a potential therapy for rare paediatric disease