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Orphan designation: Recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein (lanadelumab) for: Treatment of hereditary angioedema
Date of designation: 09/10/2015, Positive, Last updated: 04/10/2022 -
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Orphan designation: Chimeric anti-interleukin-6 monoclonal antibody (siltuximab) for: Treatment of Castleman's disease
Date of designation: 30/11/2007, Positive, Last updated: 26/08/2021 -
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Orphan designation: (2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro-1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid for: Treatment of progressive familial intrahepatic cholestasis
Date of designation: 17/07/2012, Positive, Last updated: 28/07/2021 -
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Orphan designation: Risdiplam for: Treatment of spinal muscular atrophy
Date of designation: 26/02/2019, Positive, Last updated: 04/05/2021 -
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Orphan designation: Ketoconazole for: Treatment of Cushing's syndrome
Date of designation: 23/04/2012, Positive, Last updated: 22/09/2020 -
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Orphan designation: Eculizumab for: Treatment of neuromyelitis optica spectrum disorders
Date of designation: 05/08/2013, Positive, Last updated: 20/11/2019 -
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Orphan designation: Eculizumab for: Treatment myasthenia gravis
Date of designation: 29/07/2014, 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 designation: 17/12/2010, Positive, Last updated: 20/11/2019 -
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Orphan designation: Eculizumab for: Treatment of atypical haemolytic uraemic syndrome (aHUS)
Date of designation: 24/07/2009, Positive, Last updated: 20/11/2019 -
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Orphan designation: Phosphorothioate oligonucleotide targeted to transthyretin (inotersen) for: Treatment of ATTR amyloidosis
Date of designation: 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 designation: 14/09/2007, Positive, Last updated: 26/03/2019 -
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Orphan designation: Antisense oligonucleotide targeted to the SMN2 gene (nusinersen) for: Treatment of 5q spinal muscular atrophy
Date of designation: 02/04/2012, Positive, Last updated: 16/10/2018 -
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Orphan designation: Daratumumab for: Treatment of plasma-cell myeloma
Date of designation: 17/07/2013, Positive, Last updated: 02/10/2018 -
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Orphan designation: ciclosporin for: Treatment of vernal keratoconjunctivitis
Date of designation: 06/04/2006, Positive, Last updated: 12/07/2018 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA (also known as patisiran) for: Treatment of transthyretin-mediated amyloidosis
Date of designation: 06/04/2017, Positive, Last updated: 30/05/2018 -
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Orphan designation: Budesonide for: Treatment of eosinophilic oesophagitis
Date of designation: 05/08/2013, Positive, Last updated: 18/01/2018 -
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Orphan designation: Recombinant human tripeptidyl-peptidase 1 for: Treatment of neuronal ceroid lipofuscinosis type 2
Date of designation: 12/03/2013, Positive, Last updated: 09/02/2016 -
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Orphan designation: Recombinant human nerve growth factor (cenegermin) for: Treatment of neurotrophic keratitis
Date of designation: 14/12/2015, Positive, Last updated: 02/02/2016 -
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Orphan designation: carfilzomib for: Treatment of multiple myeloma
Date of designation: 03/06/2008, Positive, Last updated: 02/12/2015 -
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Orphan designation: Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for: Treatment of chronic myeloid leukaemia
Date of designation: 02/02/2010, Positive, Last updated: 23/03/2015 -
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Orphan designation: Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for: Treatment of acute lymphoblastic leukaemia
Date of designation: 02/02/2010, Positive, Last updated: 23/03/2015 -
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Opinion/decision on a Paediatric investigation plan (PIP): Zynteglo, betibeglogene autotemcel (updated)
Decision type: RPM: decision refers to a refusal on the application for modification of an agreed PIP
Therapeutic area: Haematology-Hemostaseology
PIP number: EMEA-001665-PIP01-14-M06, Route(s) of administration: Intravenous use, Pharmaceutical form(s): Dispersion for infusion
Decision date: 13/05/2022, Last updated: 16/05/2023, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Kisplyx, Lenvatinib (updated)
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Oncology
PIP number: EMEA-001119-PIP03-19-M02, Route(s) of administration: Oral use, Pharmaceutical form(s): Capsule, hard
Decision date: 13/05/2022, Last updated: 12/05/2023, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Bylvay, Odevixibat (updated)
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Gastroentology-Hepatology
PIP number: EMEA-002054-PIP01-16-M03, Route(s) of administration: Oral use, Pharmaceutical form(s): Capsule, hard
Decision date: 13/05/2022, Last updated: 12/05/2023, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Soliris, Eculizumab
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Neurology
PIP number: EMEA-000876-PIP05-15-M05, Route(s) of administration: Intravenous use, Pharmaceutical form(s): Concentrate for solution for infusion
Decision date: 11/03/2022, Last updated: 19/04/2023, Compliance check: X