title: Duchenne muscular dystrophy drugs face tough path to approval.
abstract: Highly anticipated as new disease-modifying treatments for Duchenne muscular dystrophy (DMD), therapeutics by BioMarin Pharmaceutical (Kyndrisa™; drisapersen) and Sarepta Therapeutics (eteplirsen; AVI-4658) both recently received negative FDA reviews and are now facing battles for approval in the U.S. At present, BioMarin is committed to working with the FDA to forge a pathway to approval following the failure of its NDA, while Sarepta awaits the formal decision on its NDA, which is expected by late May 2016. Despite the critical nature of both reviews, analysts consider that there is still a narrow possibility of approval of both drugs. According to Consensus forecasts from Thomson Reuters Cortellis for Competitive Intelligence, Kyndrisa is forecast to achieve sales of USD 533.71 million in 2021.
authors.affiliation: Thomson Reuters, London, UK. lisa.hodgkinson@thomsonreuters.com.
authors.forename: L
authors.initials: L
authors.lastname: Hodgkinson
authors.affiliation: Thomson Reuters, Barcelona, Spain.
authors.forename: L
authors.initials: L
authors.lastname: Sorbera
authors.affiliation: Thomson Reuters, Barcelona, Spain.
authors.forename: A I
authors.initials: AI
authors.lastname: Graul
mesh_terms: D017277:Drug Approval; D006801:Humans; D060172:Morpholinos; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy*; D009841:Oligonucleotides / Q000627:therapeutic use*; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D060172:Morpholinos; D009841:Oligonucleotides; C525434:PRO051; C000611335:eteplirsen
keywords: Drisapersen; Duchenne muscular  dystrophy; eteplirsen