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Orphan designation: 2,2'-{2-[(1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid for: Treatment of multiple myeloma
Date of designation: 27/09/2011, Positive, Last updated: 26/08/2020 -
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Orphan designation: Ex-vivo-expanded autologous human corneal epithelium-containing stem cells for: Corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns
Date of designation: 07/11/2008, Positive, Last updated: 23/06/2020 -
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Orphan designation: Metreleptin for: Treatment of familial partial lipodystrophy
Date of designation: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Metreleptin for: Treatment of Barraquer-Simons syndrome
Date of designation: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Metreleptin for: Treatment of Lawrence syndrome
Date of designation: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Metreleptin for: Treatment of Berardinelli-Seip syndrome
Date of designation: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: tafamidis for: Treatment of senile systemic amyloidosis
Date of designation: 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 designation: 04/12/2008, Positive, Last updated: 20/11/2019 -
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Orphan designation: Lonafarnib for: Treatment of Hutchinson-Gilford progeria
Date of designation: 14/12/2018, Positive, Last updated: 01/08/2019 -
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Orphan designation: (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine for: Treatment of gastrointestinal stromal tumours
Date of designation: 17/07/2017, Positive, Last updated: 22/05/2019 -
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Orphan designation: Recombinant human alpha-Mannosidase (velmanase alfa) for: Treatment of alpha-mannosidosis
Date of designation: 26/01/2005, Positive, -
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Orphan designation: Chenodeoxycholic acid for: Treatment of inborn errors in primary bile acid synthesis
Date of designation: 16/12/2014, Positive, Last updated: 29/06/2017 -
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Orphan designation: defibrotide for: Treatment of hepatic veno-occlusive disease
Date of designation: 29/07/2004, Positive, Last updated: 17/08/2016 -
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Orphan designation: idebenone for: Treatment of Leber's hereditary optic neuropathy
Date of designation: 16/02/2007, Positive, Last updated: 11/04/2016 -
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Orphan designation: [Nle4, D-Phe7]-alfa-melanocyte stimulating hormone (afamelanotide) for: Treatment of erythropoietic protoporphyria
Date of designation: 08/05/2008, Positive, Last updated: 11/04/2016 -
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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: 3-[5-(2-Fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid (ataluren) for: Treatment of Duchenne muscular dystrophy
Date of designation: 27/05/2005, Positive, Last updated: 10/03/2015 -
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Orphan designation: (R)-2-Methyl-6-nitro-2-{4-[4-(4-trifluoromethoxyphenoxy)piperidin-1-yl]phenoxymethyl}-2,3-dihydroimidazo[2,1-b]oxazole (delamanid) for: Treatment of tuberculosis
Date of designation: 02/02/2008, Positive, Last updated: 18/06/2014 -
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Orphan designation: (1R,2S) 6-bromo-alpha-[2-(dimethylamino)ethyl]-2-methoxy-alpha-(1-naphthyl)-beta-phenyl-3-quinolineethanol bedaquiline for: Treatment of tuberculosis
Date of designation: 26/08/2005, Positive, Last updated: 01/04/2014 -
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Opinion/decision on a Paediatric investigation plan (PIP): Idefirix, Imlifidase (updated)
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Immunology-Rheumatology-Transplantation
PIP number: EMEA-002183-PIP01-17-M01, Route(s) of administration: Intravenous use, Pharmaceutical form(s): Powder for concentrate for solution for infusion
Decision date: 11/03/2022, Last updated: 23/03/2023, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Adakveo, Crizanlizumab (updated)
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Haematology-Hemostaseology
PIP number: EMEA-002141-PIP01-17-M03, Route(s) of administration: Intravenous use, Pharmaceutical form(s): Concentrate for solution for infusion
Decision date: 09/12/2021, Last updated: 22/03/2023, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Evkeeza, Evinacumab (updated)
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Endocrinology, -gynacology-fertility-metabolism
PIP number: EMEA-002298-PIP01-17-M03, Route(s) of administration: Intravenous use, Pharmaceutical form(s): Concentrate for solution for infusion
Decision date: 03/12/2021, Last updated: 22/03/2023, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Dovprela (previously Pretomanid FGK), Pretomanid (updated)
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Infectious diseases
PIP number: EMEA-002115-PIP01-17-M04, Route(s) of administration: Oral use, Pharmaceutical form(s): Dispersible tablet, Tablet
Decision date: 03/12/2021, Last updated: 21/03/2023, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Lamzede, velmanase alfa (updated)
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Endocrinology, -gynacology-fertility-metabolism
PIP number: EMEA-001056-PIP02-12-M01, Route(s) of administration: Intravenous use, Pharmaceutical form(s): Powder for solution for injection
Decision date: 23/10/2020, Last updated: 10/03/2023, Compliance check: V, 23/04/2021 -
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Opinion/decision on a Paediatric investigation plan (PIP): Retsevmo, Selpercatinib
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Oncology
PIP number: EMEA-002544-PIP01-18-M01, Route(s) of administration: Oral use, Pharmaceutical form(s): Capsule, hard, Age-appropriate dosage form
Decision date: 30/09/2021, Last updated: 13/01/2023, Compliance check: X