(Reuters) -The U.S. Food and Drug Administration has asked for a resubmission of Stealth BioTherapeutics\u2019 marketing application for its experimental therapy targeting a rare genetic condition, Barth syndrome, the company said on Thursday.<\/p>\n
The drug developer added that it has implemented a 30% workforce reduction to conserve resources for a potential resubmission.<\/p>\n
The federal health regulator\u2019s decision marks another setback in Stealth\u2019s prolonged effort to bring the therapy, elamipretide, to market after several delays.<\/p>\n
Stealth received the FDA\u2019s so-called \u2018complete response letter\u2019 after a 16.5-month review, during which the FDA extended its review from January to April to assess additional data. Last month, the regulator missed the extended deadline.<\/p>\n
The Needham, Massachusetts-based company is seeking approval for elamipretide, a potential treatment for Barth syndrome \u2013 a condition that typically affects boys and causes heart, muscle weakness and delays growth.<\/p>\n
Stealth had submitted data from a mid-stage trial, which showed improvement in knee muscle strength, which was correlated with improvements on the six-minute walk test.<\/p>\n
The FDA has agreed to consider knee extensor muscle strength, for which the company previously submitted data, as a goal to support accelerated approval, the company said.<\/p>\n
However, the regulator is hesitant to extend the accelerated pathway to critically ill newborns, who make up nearly two-thirds of the therapy\u2019s expanded access program participants.<\/p>\n
Barth syndrome affects fewer than 150 individuals in the United States and fewer than 300 worldwide, and currently, no approved therapies exist.<\/p>\n
(Reporting by Sriparna Roy in Bengaluru; Editing by Tasim Zahid)<\/p>\n","protected":false},"excerpt":{"rendered":"(Reuters) -The U.S. Food and Drug Administration has asked for a resubmission of Stealth BioTherapeutics\u2019 marketing application for…\n","protected":false},"author":2,"featured_media":148561,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[3846],"tags":[267,70,16,15],"class_list":{"0":"post-148560","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\/114606232544251865","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/148560","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=148560"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/148560\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media\/148561"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media?parent=148560"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/categories?post=148560"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/tags?post=148560"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}