{"id":60409,"date":"2025-04-29T14:19:12","date_gmt":"2025-04-29T14:19:12","guid":{"rendered":"https:\/\/www.europesays.com\/uk\/60409\/"},"modified":"2025-04-29T14:19:12","modified_gmt":"2025-04-29T14:19:12","slug":"amid-fda-chaos-approval-of-a-rare-disease-drug-gets-delayed-again","status":"publish","type":"post","link":"https:\/\/www.europesays.com\/uk\/60409\/","title":{"rendered":"Amid FDA chaos, approval of a rare disease drug gets delayed \u2014 again"},"content":{"rendered":"<p class=\"author-bio\">Ed Silverman, a senior writer and Pharmalot columnist at STAT, has been covering the pharmaceutical industry for nearly three decades.<\/p>\n<p>File this under \u201cSo close, yet so far.\u201d<\/p>\n<p>After several years of struggling with regulatory hurdles to win approval for its rare disease drug, Stealth BioTherapeutics had expected the U.S. Food and Drug Administration to respond on Tuesday to its marketing application.<\/p>\n<p>But late last week, the company received a letter saying there was a delay. Moreover, the agency did not indicate when it may now complete its review for the drug, which is called elamipretide and was developed to combat <a href=\"https:\/\/www.statnews.com\/pharmalot\/2023\/12\/18\/barth-syndrome-stealth-biotherapeutics-elamipretide-rare-disease-fda\/\" target=\"_blank\" rel=\"noopener\">Barth syndrome<\/a>. The rare illness, which causes an enlarged heart, muscle weakness and a shortened life expectancy, afflicts nearly 150 people in the U.S.<\/p>\n<p class=\"restricted-text\">STAT+ Exclusive Story<\/p>\n<p class=\"restricted-login\">Already have an account? <a href=\"https:\/\/www.statnews.com\/login\/\" data-stat-login=\"\" data-stat-paywall-cta=\"breaker login cta\" target=\"_blank\" rel=\"noopener\">Log in<\/a><\/p>\n<p>\t\t\t<img decoding=\"async\" class=\"restricted-content-breaker-bg\" src=\"https:\/\/www.europesays.com\/uk\/wp-content\/uploads\/2025\/04\/stat_plus_hero_new-2048x502.png\" alt=\"STAT+\"\/><\/p>\n<p>\t\t\t\t\t<a href=\"https:\/\/www.statnews.com\/stat-plus\/\" target=\"_blank\" rel=\"noopener\"><br \/>\n\t\t\t\t\t\t<img decoding=\"async\" class=\"stat-plus-logo\" src=\"https:\/\/www.europesays.com\/uk\/wp-content\/uploads\/2025\/04\/stat-plus-logo-white.svg.svg+xml\" alt=\"STAT+\"\/><br \/>\n\t\t\t\t\t<\/a><\/p>\n<p>\t\t\t\tThis article is exclusive to STAT+ subscribers<br \/>\n\t\t\t\tUnlock this article \u2014 plus in-depth analysis, newsletters, premium events, and news alerts.<\/p>\n<p class=\"restricted-content-breaker-wrapper-cta mobile\">Already have an account? <a href=\"https:\/\/www.statnews.com\/login\/\" data-stat-login=\"\" data-stat-paywall-cta=\"breaker login cta mobile\" target=\"_blank\" rel=\"noopener\">Log in<\/a><\/p>\n<p>\t\t\t\t\t\tIndividual plans<\/p>\n<p>\t\t\t\t\t\tGroup plans<\/p>\n<p>\t\t\t\t<a href=\"https:\/\/www.statnews.com\/stat-plus\/\" class=\"stat-button view-all\" data-stat-paywall-cta=\"breaker view all plans\" aria-label=\"View All Plans\" target=\"_blank\" rel=\"noopener\">View All Plans<\/a><\/p>\n<p class=\"restricted-content-text\">\n\t\t\t\t\t\tTo read the rest of this story subscribe to STAT+.\t\t\t\t\t<\/p>\n<p>\t\t\t\t\t<a href=\"https:\/\/www.statnews.com\/stat-plus\/\" class=\"stat-button restricted-button\" data-stat-paywall-cta=\"breaker subscribe cta 1\" target=\"_blank\" rel=\"noopener\">Subscribe<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"Ed Silverman, a senior writer and Pharmalot columnist at STAT, has been covering the pharmaceutical industry for nearly&hellip;\n","protected":false},"author":2,"featured_media":60410,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[4315],"tags":[12848,5885,2460,105,4326,5489,46,6448,31173,16,15],"class_list":{"0":"post-60409","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-medication","8":"tag-biotechnology","9":"tag-drug-development","10":"tag-fda","11":"tag-health","12":"tag-medication","13":"tag-pharmaceuticals","14":"tag-policy","15":"tag-rare-disease","16":"tag-stat","17":"tag-uk","18":"tag-united-kingdom"},"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@uk\/114421692232796831","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/60409","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=60409"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/60409\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media\/60410"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media?parent=60409"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/categories?post=60409"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/tags?post=60409"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}