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Orphan designation: miglustat for: Treatment of Niemann-Pick's disease, type C
Date of designation: 16/02/2006, Expired, Last updated: 20/02/2019 -
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Orphan designation: sildenafil citrate for: Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension
Date of designation: 12/12/2003, Expired, Last updated: 19/01/2016 -
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Orphan designation: Rufinamide for: Treatment of Lennox-Gastaut syndrome
Date of designation: 20/10/2004, Expired, Last updated: 20/02/2019 -
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Orphan designation: azacitidine for: Treatment of myelodysplastic syndromes
Date of designation: 06/02/2002, Expired, Last updated: 20/02/2019 -
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Orphan designation: 1, 1'-[1,4-Phenylenebis (methylene)]-bis-1,4,8,11-tetraazacyclotetradecane (plerixafor) for: Treatment to mobilize progenitor cells prior to stem cell transplantation
Date of designation: 20/10/2004, Expired, Last updated: 10/08/2021 -
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Orphan designation: Ibuprofen for: Treatment of patent ductus arteriosus
Date of designation: 15/02/2001, Expired, Last updated: 06/03/2015 -
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Orphan designation: Refusal of orphan designation for the treatment of poisoning by local anaesthetics
Date of refusal of designation: 03/04/2017, Negative, Last updated: 15/05/2017 -
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Orphan designation: Refusal of orphan designation for the treatment of narcolepsy
Date of refusal of designation: 13/07/2016, Negative, Last updated: 09/01/2017 -
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Orphan designation: Refusal of orphan designation for the treatment of fibromyalgia
Date of refusal of designation: 06/10/2016, Negative, Last updated: 13/12/2016 -
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Orphan designation: Refusal of orphan designation for the treatment of uraeumic pruritus
Date of refusal of designation: 27/02/2015, Negative, Last updated: 02/10/2015 -
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Orphan designation: Refusal of orphan designation for the treatment of non-small cell lung cancer that is anaplastic lymphoma kinase (ALK) positive
Date of refusal of designation: 28/03/2014, Negative, Last updated: 10/03/2015 -
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Orphan designation: Refusal of orphan designation for the treatment of autosomal dominant polycystic kidney disease
Date of refusal of designation: 11/12/2014, Negative, Last updated: 10/03/2015 -
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Orphan designation: Refusal of orphan designation for the treatment of complex regional pain syndrom
Date of refusal of designation: 17/04/2013, Negative, Last updated: 23/04/2013 -
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Orphan designation: Refusal of orphan designation for the treatment of P-gp positive breast cancer
Date of refusal of designation: 21/03/2012, Negative, Last updated: 06/09/2012 -
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Orphan designation: Refusal of orphan designation for the treatment of off-periods in adult patients with advanced Parkinson's disease
Date of refusal of designation: 09/09/2010, Negative, Last updated: 30/03/2012 -
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Orphan designation: Refusal of orphan designation for the treatment of amyotropic lateral sclerosis
Date of refusal of designation: 16/01/2011, Negative, Last updated: 20/12/2011 -
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Orphan designation: Refusal of orphan designation for the treatment of cystic fibrosis
Date of refusal of designation: 16/07/2010, Negative, Last updated: 09/08/2010 -
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Orphan designation: Refusal of orphan designation for the treatment of pancreatic cancer
Date of refusal of designation: 16/07/2010, Negative, Last updated: 05/08/2010 -
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Orphan designation: Refusal of orphan designation for the prevention of graft rejection after liver transplantation
Date of refusal of designation: 10/09/2008, Negative, Last updated: 30/10/2009 -
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Orphan designation: Refusal of orphan designation for the treatment of B-cell non-Hodgkin's lymphoma
Date of refusal of designation: 01/02/2002, Negative, Last updated: 17/09/2009 -
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Orphan designation: Refusal of orphan designation for the treatment of pancreatic cancer
Date of refusal of designation: 04/12/2007, Negative, Last updated: 29/07/2008 -
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Orphan designation: Refusal of orphan designation for the treatment of painful HIV-associated neuropathy
Date of refusal of designation: 21/09/2006, Negative, Last updated: 19/07/2007 -
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Orphan designation: Refusal of orphan designation for the treatment of painful HIV-associated neuropathy
Date of refusal of designation: 21/09/2006, Negative, Last updated: 19/07/2007 -
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Orphan designation: Refusal of orphan designation for the treatment of status epilepticus
Date of refusal of designation: 01/03/2004, Negative, Last updated: 12/12/2005 -
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Orphan designation: Refusal of orphan designation for the treatment of active phase of Peyronie's disease
Date of refusal of designation: 16/06/2005, Negative, Last updated: 27/10/2005