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48 results
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Summary of opinion: Evrysdi
risdiplam, opinion date: 25/02/2021, Positive, Last updated: 26/02/2021 -
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Summary of opinion: Cabometyx
cabozantinib, opinion date: 25/02/2021, Positive, Last updated: 26/02/2021 -
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Summary of opinion: Bluevac BTV (previously known as Bluevac BTV8)
opinion date: 18/06/2020, Positive, Last updated: 19/06/2020 -
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Referral: Lartruvo
olaratumab, associated names: Lartruvo, Article 20 procedures
Status: European Commission final decision, opinion/position date: 31/01/2019, EC decision date: 19/07/2019, Last updated: 22/08/2019 -
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Referral: Factor VIII
associated names: Iblias, Elocta, Kovaltry, Nuwiq, Obizur, NovoEight, Voncento, ReFacto AF, Kogenate Bayer, Helixate NexGen, Advate, Article 31 referrals
Status: European Commission final decision, opinion/position date: 14/09/2017, EC decision date: 10/10/2017, Last updated: 05/12/2017 -
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Referral: Direct-acting antivirals indicated for treatment of hepatitis C (interferon-free)
daclatasvir / dasabuvir / sofosbuvir / ledipasvir / simeprevir / ombitasvir/paritaprevir / ritonavir, associated names: Sovaldi, Olysio, Viekirax, Harvoni, Exviera, Daklinza, Article 20 procedures
Status: European Commission final decision, opinion/position date: 15/12/2016, EC decision date: 23/02/2017, Last updated: 09/03/2017 -
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Orphan designation: recombinant human interleukin-3 truncated diphtheria toxin fusion protein for: Treatment of blastic plasmacytoid dendritic cell neoplasm
Date of first decision: 11/11/2015, Positive, Last updated: 25/01/2021 -
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Orphan designation: Obiltoxaximab for: Treatment of anthrax
Date of first decision: 24/08/2018, Positive, Last updated: 27/11/2020 -
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Orphan designation: Recombinant human beta-glucuronidase (vestronidase alfa) for: Treatment of mucopolysaccharidosis type VII (Sly syndrome)
Date of first decision: 21/03/2012, Positive, Last updated: 11/11/2020 -
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Orphan designation: N-(2,4-di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide (ivacaftor) for: Treatment of cystic fibrosis
Date of first decision: 08/07/2008, Positive, Last updated: 26/10/2020 -
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Orphan designation: Ketoconazole for: Treatment of Cushing's syndrome
Date of first decision: 23/04/2012, Positive, Last updated: 22/09/2020 -
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Orphan designation: Metreleptin for: Treatment of familial partial lipodystrophy
Date of first decision: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Metreleptin for: Treatment of Barraquer-Simons syndrome
Date of first decision: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Metreleptin for: Treatment of Lawrence syndrome
Date of first decision: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Metreleptin for: Treatment of Berardinelli-Seip syndrome
Date of first decision: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate (tafamidis) for: Treatment of familial amyloid polyneuropathy
Date of first decision: 28/08/2006, Positive, Last updated: 25/02/2020 -
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Orphan designation: tafamidis for: Treatment of senile systemic amyloidosis
Date of first decision: 08/11/2012, Positive, Last updated: 25/02/2020 -
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Orphan designation: Recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein for: Treatment of hypophosphatasia
Date of first decision: 04/12/2008, Positive, Last updated: 20/11/2019 -
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Orphan designation: Recombinant human lysosomal acid lipase for: Treatment of lysosomal acid lipase deficiency
Date of first decision: 17/12/2010, Positive, Last updated: 20/11/2019 -
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Orphan designation: Eculizumab for: Treatment myasthenia gravis
Date of first decision: 29/07/2014, Positive, Last updated: 20/11/2019 -
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Orphan designation: Eculizumab for: Treatment of neuromyelitis optica spectrum disorders
Date of first decision: 05/08/2013, Positive, Last updated: 20/11/2019 -
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Orphan designation: Eculizumab for: Treatment of atypical haemolytic uraemic syndrome (aHUS)
Date of first decision: 24/07/2009, Positive, Last updated: 20/11/2019 -
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Orphan designation: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene for: Treatment of beta thalassaemia intermedia and major
Date of first decision: 24/01/2013, Positive, Last updated: 03/06/2019 -
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Orphan designation: Phosphorothioate oligonucleotide targeted to transthyretin (inotersen) for: Treatment of ATTR amyloidosis
Date of first decision: 26/03/2014, Positive, Last updated: 17/05/2019 -
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Orphan designation: Human coagulation factor X for: Treatment of hereditary factor X deficiency
Date of first decision: 14/09/2007, Positive, Last updated: 26/03/2019