{"id":36269,"date":"2022-06-04T09:53:53","date_gmt":"2022-06-04T09:53:53","guid":{"rendered":"https:\/\/harchi90.com\/another-refuse-to-file-for-a-small-biotech-is-the-fda-using-rtfs-to-manage-its-workload-endpoints-news\/"},"modified":"2022-06-04T09:53:53","modified_gmt":"2022-06-04T09:53:53","slug":"another-refuse-to-file-for-a-small-biotech-is-the-fda-using-rtfs-to-manage-its-workload-endpoints-news","status":"publish","type":"post","link":"https:\/\/harchi90.com\/another-refuse-to-file-for-a-small-biotech-is-the-fda-using-rtfs-to-manage-its-workload-endpoints-news\/","title":{"rendered":"Another refuse-to-file for a small biotech – is the FDA using RTFs to manage its workload? – Endpoints News"},"content":{"rendered":"
\n

As the regular flow of NDA and BLA submissions continues to pick up, it’s little wonder that an already Covid-beleaguered FDA seems to be increasingly turning to refuse-to-file letters as a means to provide instructive feedback, and perhaps even to slow the pace of its workload.<\/p>\n

The latest RTF letter, disclosed Thursday by Aeglea BioTherapeutics, a penny-stock, clinical-stage biotech company calls for new efficacy data to be included in its BLA for pegzilarginase for the treatment of the rare, progressive and debilitating disease characterized by high levels of arginine, and known as Arginase 1 Deficiency. The company’s stock price fell by more than 40% on Thursday.<\/p>\n

Unlike complete response letters, which are essentially FDA rejections, RTFs allow the FDA to reject an application before beginning a more complete review. But some tracking the industry closely think the FDA has been turning more and more to these RTFs, and in instances where the agency may have seemed to be less than forthcoming, or so the companies say.<\/p>\n

Nymox Pharmaceutical disclosed an RTF last week and similarly saw its share price plummet. In Nymox’s case, the company says it wasn’t informed by the FDA of what was coming.<\/p>\n

The FDA requested longer-term safety data in its NDA for fexapotide triflutate as a potential treatment for benign prostatic hyperplasia or prostate enlargement. The company said longer-term safety data (as long as 6 years after a single low dose non-systemic injection given one time only) “was not requested by the FDA in any previous pre-NDA communications.”<\/p>\n

But companies aren’t always forthcoming in disclosing what the FDA-cited deficiencies are in their RTF letters.<\/p>\n

A review published in JAMA last year of RTF letters from 2008 to 2017 found that 85% of the FDA’s refusals (544 of 644) were due to scientific deficiencies; most of which were related to drug efficacy and safety (30%) and drug quality (19%).<\/p>\n

But the researchers found that applicants publicly disclosed the issuance of only 16% of RTF letters (16 of 103) and disclosed only 5% of the refusal reasons (35 of 644) that the FDA cited in its letters.<\/p>\n

And RTF letters are still rare – only amounting to 4% of the new applications and efficacy supplements FDA received over the study period.<\/p>\n

In Aeglea’s case, the company seemed to indicate that the agency was clear that it’s requesting more “evidence showing that plasma arginine and metabolite reduction predicts clinical benefit in patients with ARG1-D or clinical data demonstrating a treatment effect on clinically meaningful outcomes.”<\/p>\n

Peter Marks, FDA<\/span><\/div>\n

The FDA also requested additional CMC data, and Aeglea said it intends to request a Type A meeting (ie for stalled development programs) with the FDA to clarify and respond to the RTF letter.<\/p>\n

Those interactions are key as CBER’s Peter Marks recently indicated at a conference that his center is relying mostly on written communications to talk with sponsors.<\/p>\n

Marks acknowledged CBER’s staff shortages, which should be ameliorated somewhat by the incoming user fee deals, and that this \u201cis not an optimal situation. Even prior to the pandemic, we were understaffed. My key for 2022 is a recovery theme – my hope is that as we ease into the next calendar year, more robust exchanges with the agency occur, including teleconferences. “<\/p>\n<\/p><\/div>\n","protected":false},"excerpt":{"rendered":"

As the regular flow of NDA and BLA submissions continues to pick up, it’s little wonder that an already Covid-beleaguered FDA seems to be increasingly turning to refuse-to-file letters as a means to provide instructive feedback, and perhaps even to slow the pace of its workload. The latest RTF letter, disclosed Thursday by Aeglea BioTherapeutics, …<\/p>\n

Another refuse-to-file for a small biotech – is the FDA using RTFs to manage its workload? – Endpoints News<\/span> Read More »<\/a><\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"site-sidebar-layout":"default","site-content-layout":"default","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"","ast-breadcrumbs-content":"","ast-featured-img":"","footer-sml-layout":"","theme-transparent-header-meta":"","adv-header-id-meta":"","stick-header-meta":"","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","spay_email":"","jetpack_publicize_message":"","jetpack_is_tweetstorm":false,"jetpack_publicize_feature_enabled":true},"categories":[8],"tags":[],"jetpack_featured_media_url":"","jetpack_publicize_connections":[],"jetpack_sharing_enabled":true,"fifu_image_url":"https:\/\/endpts.com\/wp-content\/uploads\/2022\/02\/FDA-AP-social-scaled.jpg","_links":{"self":[{"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/posts\/36269"}],"collection":[{"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/comments?post=36269"}],"version-history":[{"count":0,"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/posts\/36269\/revisions"}],"wp:attachment":[{"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/media?parent=36269"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/categories?post=36269"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/tags?post=36269"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}