{"id":4534,"date":"2025-05-15T13:54:43","date_gmt":"2025-05-15T04:54:43","guid":{"rendered":"https:\/\/bionauts.jp\/?p=4534"},"modified":"2025-05-26T17:02:06","modified_gmt":"2025-05-26T08:02:06","slug":"therapeutic-drug-for-achondroplasia","status":"publish","type":"post","link":"https:\/\/bionauts.jp\/?p=4534","title":{"rendered":"Therapeutic Drug for Achondroplasia"},"content":{"rendered":"<h3>Advantages<\/h3>\n<ul>\n<li>Oral administration possible.<\/li>\n<li>High safety.<\/li>\n<li>Improvement of some indicators confirmed in investigator-initiated Phase 2 clinical trial.<\/li>\n<\/ul>\n<h3>Current Stage and Key Data<\/h3>\n<p><u>Investigator-initiated clinical trial Phase 2<\/u><\/p>\n<ul>\n<li>Safety has been tested in investigator-initiated clinical trial Phase 1.<\/li>\n<li>Improvement in some indicators has been confirmed in Phase 2 investigator-initiated clinical trial (multicenter open-label, 9 patients, meclizine administered once daily for 26 weeks). Detailed data can be disclosed under CDA.<\/li>\n<li>Efficacy, PK\/PD, and safety have been confirmed in animal experiments (see references).<\/li>\n<\/ul>\n<table style=\"border-collapse: collapse; width: 73.5986%; height: 525px;\">\n<tbody>\n<tr>\n<td style=\"width: 100%;\"><a href=\"http:\/\/bionauts.jp\/wp-content\/uploads\/2025\/05\/BK-03867.jpg\"><img decoding=\"async\" loading=\"lazy\" class=\"alignleft size-full wp-image-4535\" src=\"http:\/\/bionauts.jp\/wp-content\/uploads\/2025\/05\/BK-03867.jpg\" alt=\"\" width=\"518\" height=\"600\" srcset=\"https:\/\/bionauts.jp\/wp-content\/uploads\/2025\/05\/BK-03867.jpg 518w, https:\/\/bionauts.jp\/wp-content\/uploads\/2025\/05\/BK-03867-259x300.jpg 259w\" sizes=\"(max-width: 518px) 85vw, 518px\" \/><\/a><\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n<h3>Partnering Model<\/h3>\n<p><u>Seeking partner companies to collaborate on further clinical development and approval applications.<\/u><\/p>\n<ul>\n<li>Examples of potential partners: Biotech\/pharmaceutical companies focusing on repositioning and development of treatments for orthopedics and rare diseases.<\/li>\n<\/ul>\n<h3>Background<\/h3>\n<p>Achondroplasia is a congenital disease that causes short limbs and short stature (final height: 120-130 cm), with a prevalence of about 5 cases per 100,000. It is caused by enhanced signaling due to a gain-of-function mutation in FGFR3. The current standard treatment is growth hormone and limb lengthening surgery, but these are not effective enough. In recent years, vosoritide has been approved as a new therapeutic drug that suppresses FGFR3 signaling (peptide formulation, daily subcutaneous injection). However, the burden on patients remains large, and new therapeutic drugs are needed.<\/p>\n<h3>Principal Investigator<\/h3>\n<p>Masaki MATSUSHITA (Department of Orthopedics\/Rheumatology, Nagoya University Graduate School of Medicine, Tokai Higher Education and Research System)<\/p>\n<h3>References<\/h3>\n<ul>\n<li>Papers: <a href=\"https:\/\/www.ncbi.nlm.nih.gov\/pmc\/articles\/PMC5547068\/\" target=\"_blank\" rel=\"noopener\">Matsushita et al. (2017) <\/a><a href=\"https:\/\/www.ncbi.nlm.nih.gov\/pmc\/articles\/PMC5547068\/\"><em>Sci Rep. <\/em><\/a><a href=\"https:\/\/www.ncbi.nlm.nih.gov\/pmc\/articles\/PMC5547068\/\">7;7(1):7371 <\/a><\/li>\n<li>Patents: JP Patent No. 6232630<\/li>\n<\/ul>\n<p>&nbsp;<\/p>\n<p>Project ID:BK-03867<\/p>\n","protected":false},"excerpt":{"rendered":"<p>The approved antihistamine meclizine promotes cartilage formation by suppressing excessive signaling caused by FGFR3 mutations.<\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_mi_skip_tracking":false,"_monsterinsights_sitenote_active":false,"_monsterinsights_sitenote_note":"","_monsterinsights_sitenote_category":0},"categories":[1],"tags":[],"categories_of_inventions":[],"disease_categories___indication":[],"asset_type":[],"development_phase":[],"medicine":[101,96],"other_than_medicine":[],"material":[],"engineering":[],"software_it":[],"aioseo_notices":[],"_links":{"self":[{"href":"https:\/\/bionauts.jp\/index.php?rest_route=\/wp\/v2\/posts\/4534"}],"collection":[{"href":"https:\/\/bionauts.jp\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/bionauts.jp\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=4534"}],"version-history":[{"count":4,"href":"https:\/\/bionauts.jp\/index.php?rest_route=\/wp\/v2\/posts\/4534\/revisions"}],"predecessor-version":[{"id":4539,"href":"https:\/\/bionauts.jp\/index.php?rest_route=\/wp\/v2\/posts\/4534\/revisions\/4539"}],"wp:attachment":[{"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=4534"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=4534"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=4534"},{"taxonomy":"categories_of_inventions","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories_of_inventions&post=4534"},{"taxonomy":"disease_categories___indication","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fdisease_categories___indication&post=4534"},{"taxonomy":"asset_type","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fasset_type&post=4534"},{"taxonomy":"development_phase","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fdevelopment_phase&post=4534"},{"taxonomy":"medicine","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fmedicine&post=4534"},{"taxonomy":"other_than_medicine","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fother_than_medicine&post=4534"},{"taxonomy":"material","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fmaterial&post=4534"},{"taxonomy":"engineering","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fengineering&post=4534"},{"taxonomy":"software_it","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fsoftware_it&post=4534"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}