Expanded access as a source of real-world data: An overview of FDA and EMA approvals 97 5♦ SUPPLEMENTARY MATERIAL Supplementary Table 1: Overview of FDA and EMA approvals (partly) based on expanded access efficacy data Generic name Sponsor Pivotal9 Efficacy10 FDA11 EMA abacavir ViiV Healthcare BV no FDA/EMA 1998 1999 afamelanotide Clinuvel no EMA N 2014 alglucosidase alfa Genzyme no FDA/EMA 2006 2006 amphotericin ꞵ Astellas FDA FDA 2000 N anagrelide Shire Pharmaceuticals EMA FDA/EMA 1997 2004 autologous CD34+ enriched cell fraction Orchard Therapeutics no EMA N 2016 aztreonam lysine Gilead Sciences no FDA 2010 2009 blinatumomab Amgen no FDA 2014 2015 bosutinib Pfizer no EMA 2012 2013 caspofungin Accord Healthcare no FDA/EMA 2001 2001 cholic acid Retrophin FDA/EMA FDA/EMA 2015 2015 clarithromycin Abbott Pharmaceuticals FDA FDA 1993 N clofarabine Genzyme no FDA 2004 2006 daclatasvir dihydrochloride Bristol-Myers Squibb no FDA 2015 2014 defibrotide Gentium no FDA 2016 2013 dinutuximab ꞵ EUSA Pharma EMA EMA N 2017 fish oil triglycerides Fresenius Kabi FDA FDA 2018 N glucarpidase BTG FDA FDA 2012 W idebenone Santhera Pharmaceuticals no EMA N 2015 imatinib Novartis no FDA/EMA 2001 2001 isavuconazole Basilea Pharmaceutica no EMA 2015 2015 ivermectin Merck Sharp Dohme no FDA 1996 N lamivudine GlaxoSmithKline no EMA 1998 1999 linezolid Pharmacia & Upjohn no FDA 2000 N lutetium (177Lu) oxodotreotide Advanced Accelerator Applications FDA/EMA FDA/EMA 2017 2017 mercaptamine hydrochloride Recordati Rare Diseases no EMA 2012 2017 miltefosine Knight Therapeutics no FDA 2014 N nitisinone Swedish Orphan Biovitrum FDA/EMA FDA/EMA 2002 2005 pitolisant Bioprojet Pharma no EMA N 2016 propranolol hydrochloride Pierre Fabre Dermatologie no FDA/EMA 2014 2014 9 Pivotal depicts if FDA/EMA used data from EA in a pivotal way to support the efficacy profile. 10 Determines whether FDA and or EMA used data from EA to support the efficacy profile. 11 Year of EMA/FDA approval (if applicable). W: Withdrawn, N: Not approved.
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