industry biotechnology founded 2002 headquarters leiden netherlands key people hans schikan ceo berndt modig cfo giles champion cmo luc dochez cbo david mott chairman products pharmaceuticals number of employees 89 2014 website www.prosensa.com prosensa was a biotechnology company engaged in the discovery development and commercialization of rna modulating therapeutics . the company targets genetic disorders with a large unmet medical need with a primary focus on neuromuscular and neurodegenerative disorders such as duchenne muscular dystrophy dmd myotonic dystrophy and huntington s disease . prosensa was acquired by biomarin 1 contents 1 history up to the sale 2 related events since the sale 3 external links 4 references history up to the sale edit prosensa commenced operations in 2002 and is located in leiden the netherlands . the company works closely together with academia patient groups and experts worldwide . prosensa entered into an exclusive licensing agreement in 2003 with leiden university medical center lumc for their proprietary rna modulation exon skipping technology to develop treatments for dmd other neuromuscular disorders and indications outside the field of neuromuscular disorders . prosensa s portfolio of clinical and pre clinical rna based drug candidates is currently focused on the treatment of dmd . prosensa s lead product drisapersen has completed phase iii clinical trials and in june 2015 a new drug application nda has been accepted by the us fda . 2 three additional products pro044 pro045 and pro053 were also in clinical development and pro052 and pro055 were in advanced preclinical development . in parallel prosensa also had an advanced program called prospect into pre clinical testing which included a new and innovative application of its exon skipping technology platform applying multiple exon skipping to specifically target rarer mutations initially in exon 10 to 30 region in the dystrophin gene . this approach could have applicability between 5 13 % of the dmd population . citation needed in addition to its clinical trials prosensa were running a natural history study which has completed enrollment with 269 patients across the us europe and latin america . the purpose of this study was to characterize the natural history and progression of dmd to help inform the design of future studies to capture biomarkers of safety and disease progression and to provide comparative data for the development of rare exons for which formal controlled trials are not feasible . in november 2014 biomarin pharmaceutical agreed to pay up to 840 million to acquire prosensa . 3 related events since the sale edit in january 2016 the fda rejected drisapersen largely on the basis of toxicity . 4 in may 2016 biomarin discontinued the clinical and regulatory development of drisapersen as well as phase 2 studies of bmn 044 bmn 045 and bmn 053 formerly pro044 pro045 and pro053 . 5 external links edit official website references edit http investors.bmrn.com releasedetail.cfm releaseid 884456 fda accepts biomarin s nda for drisapersen . parent project md. june 29 2015 . http online.wsj.com articles biomarin to pay up to 840 million for prosensa 1416826801 fda rejects biomarin s muscle wasting drug ; sarepta drug in focus . jan 2016 biomarin announces withdrawal of market authorization application for kyndrisa drisapersen in europe nasdaq bmrn . investors.bmrn.com . retrieved 2016 06 09 . retrieved from https en.wikipedia.org w index.php title prosensa & oldid 724586546 categories biotechnology companies of the netherlandscompanies listed on nasdaqcompanies established in 2002dutch brandshidden categories all articles with unsourced statementsarticles with unsourced statements from april 2016 navigation menu personal tools not logged intalkcontributionscreate accountlog in namespaces article talk variants views read edit view history more search navigation main pagecontentsfeatured contentcurrent eventsrandom articledonate to wikipediawikipedia store interaction helpabout wikipediacommunity portalrecent changescontact page tools what links hererelated changesupload filespecial pagespermanent linkpage informationwikidata itemcite this page print export create a bookdownload as pdfprintable version languages nederlands edit links this page was last modified on 10 june 2016 at 06 05 . text is available under the creative commons attribution sharealike license ; additional terms may apply . by using this site you agree to the terms of use and privacy policy . wikipedia is a registered trademark of the wikimedia foundation inc. a non profit organization . privacy policy about wikipedia disclaimers contact wikipedia developers cookie statement mobile view