{"id":103781,"date":"2025-05-15T14:57:09","date_gmt":"2025-05-15T14:57:09","guid":{"rendered":"https:\/\/www.europesays.com\/uk\/103781\/"},"modified":"2025-05-15T14:57:09","modified_gmt":"2025-05-15T14:57:09","slug":"kaerus-bioscience-secures-fda-approval-for-rare-genetic-disorder-treatment","status":"publish","type":"post","link":"https:\/\/www.europesays.com\/uk\/103781\/","title":{"rendered":"Kaerus Bioscience secures FDA approval for rare genetic disorder treatment"},"content":{"rendered":"<p>Ella Day | May 15, 2025 | News story | <a href=\"https:\/\/pharmafile.com\/sector\/research-and-development\/\" target=\"_blank\" rel=\"noopener\">Research and Development<\/a> |\u00a0\u00a0<a href=\"https:\/\/pharmafile.com\/tag\/fda\/\" target=\"_blank\" rel=\"noopener\">FDA<\/a>, <a href=\"https:\/\/pharmafile.com\/tag\/fragile-x-syndrome\/\" target=\"_blank\" rel=\"noopener\">Fragile X syndrome<\/a>, <a href=\"https:\/\/pharmafile.com\/tag\/kaerus-bioscience\/\" target=\"_blank\" rel=\"noopener\">Kaerus Bioscience<\/a>, <a href=\"https:\/\/pharmafile.com\/tag\/orphan-drug-designation-odd\/\" target=\"_blank\" rel=\"noopener\">Orphan Drug Designation (ODD)<\/a>, <a href=\"https:\/\/pharmafile.com\/therapeuticareas\/rare-diseases\/\" target=\"_blank\" rel=\"noopener\">Rare Diseases<\/a>, <a href=\"https:\/\/pharmafile.com\/tag\/rare-pediatric-drug-designations-rpdd\/\" target=\"_blank\" rel=\"noopener\">Rare Pediatric Drug Designations (RPDD)<\/a>, <a href=\"https:\/\/pharmafile.com\/tag\/genetic-syndromes\/\" target=\"_blank\" rel=\"noopener\">genetic syndromes<\/a>\u00a0<\/p>\n<p>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\u2019 aim to deliver the first effective treatment for the disorder.<\/p>\n<p>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\u2019 preclinical animal studies and data.<\/p>\n<p>\u201cThese important FDA designations are more excellent news for Kaerus coming after the impressive phase 1 results showing KER-0193,\u201d commented Paul Sekhri, chairman of Kaerus.<\/p>\n<p>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.<\/p>\n<p>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.<\/p>\n<p>Robert Ring, CEO of Kaerus, said: \u201cThe FDA\u2019s granting of ODD and RPDD for KER-0193 is an important step towards our objective of delivering an effective treatment for people with FXS.\u201d<\/p>\n<p>The company is finalising preparations for a phase 2 study in FXS patients.<\/p>\n<p>Ella Day<br \/>15\/5\/25<\/p>\n","protected":false},"excerpt":{"rendered":"Ella Day | May 15, 2025 | News story | Research and Development |\u00a0\u00a0FDA, Fragile X syndrome, Kaerus&hellip;\n","protected":false},"author":2,"featured_media":103782,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[3846],"tags":[267,70,16,15],"class_list":{"0":"post-103781","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-genetics","8":"tag-genetics","9":"tag-science","10":"tag-uk","11":"tag-united-kingdom"},"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@uk\/114512438340743845","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/103781","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/comments?post=103781"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/103781\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media\/103782"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media?parent=103781"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/categories?post=103781"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/tags?post=103781"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}