Separating fact from fiction: US regulatory timelines

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CLINUVEL’s drug SCENESSE® (afamelanotide) was approved by the US Food and Drug Administration (FDA) on 08 October 2019 for adult patients with erythropoietic protoporphyria (EPP). In June 2019 it was announced that the FDA had requested an extension of the Prescription Drug User Fee Act (PDUFA) date, which was ultimately set on 08 October 2019, when approval was granted. Dr Wolgen provided some more context on this moment in his 2019 Annual Report letter:

On 31 May, the FDA used its discretionary tool to request a three month extension to the formal review user fee goal date, providing a new target date 6 October.* We assessed the delay as a positive measure, since the Agency had had ample opportunity to reject the submission, issue a Refusal to File or even request a withdrawal. None of the events had occurred during our submission or review, and our teams kept working towards satisfying all outstanding questions from the agency. It was a period whereby many around us were tested, but the overwhelming majority of investors kept having faith in our teams. A key ingredient to CLINUVEL’s success continues to be our consistency.

* The FDA later clarified the target date as 08 October 2019, see below.

A brief history of announcements on US regulatory interactions and US clinical trials for EPP to date is provided below.

Date Announcement
29 Jul 2008 FDA grants CLINUVEL orphan-drug designation
23 Dec 2008 CLINUVEL lodges IND in US
29 Jan 2009 FDA allows IND status for CLINUVEL’s afamelanotide
30 Mar 2010 FDA allows US clinical trials of afamelanotide in EPP
Breakthrough Australian drug to commence US patient trials (media release)
09 Nov 2010 FDA provides positive guidance on CLINUVEL’s EPP program
09 Feb 2011 CLINUVEL completes first Phase II US study
03 Nov 2011 CLINUVEL demonstrates positive treatment effect of afamelanotide in US Phase II study
15 Mar 2012 CLINUVEL, FDA agree in principle to Phase III study with SCENESSE® (afamelanotide)
22 May 2012 CLINUVEL commences US Phase III trial of SCENESSE® in rare light and UV disorder
11 Nov 2013 Clinical significance and benefit from SCENESSE® in US Phase III EPP study
27 Aug 2015 FDA grants CLINUVEL a Type C Meeting
05 Oct 2015 CLINUVEL and FDA discuss EPP indication and regulatory pathways for SCENESSE®
06 Jul 2016 FDA awards SCENESSE® Fast Track designation for treatment of EPP
18 Jul 2016 FDA accepts SCENESSE® clinical data package for NDA submission
13 Oct 2016 FDA, CLINUVEL to discuss SCENESSE® at pre-NDA meeting (media release)
24 Oct 2016 FDA erythropoietic protoporphyria (EPP) scientific workshop
09 Nov 2016 CLINUVEL agrees with FDA on New Drug Application timelines
19 Sep 2017 CLINUVEL provides update on SCENESSE® FDA filing
14 May 2018 CLINUVEL regulatory update
25 Jun 2018 CLINUVEL completes SCENESSE® FDA filing
05 Sep 2018 US FDA update
10 Jan 2019 US FDA sets PDUFA date for SCENESSE®
03 Jun 2019 US FDA review for SCENESSE® extended by three months (media release)
02 Oct 2019 FDA clarifies SCENESSE® PDUFA date
09 Oct 2019 FDA grants Marketing Approval for SCENESSE®
16 Apr 2020 First US patients to be treated with SCENESSE®

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