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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: 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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Opinion/decision on a Paediatric investigation plan (PIP): Waylivra, Volanesorsen
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Endocrinology, -gynacology-fertility-metabolism
PIP number: EMEA-001915-PIP01-15-M01, Route(s) of administration: Intravenous use, Pharmaceutical form(s): Solution for injection
Decision date: 04/12/2019, Last updated: 02/04/2020, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Vosevi, Sofosbuvir, velpatasvir, voxilaprevi
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Infectious diseases
PIP number: EMEA-001822-PIP01-15-M01, Route(s) of administration: Oral use, Pharmaceutical form(s): Film-coated tablet, Age-appropriate oral solid dosage form
Decision date: 06/01/2020, Last updated: 12/06/2020, Compliance check: X -
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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 first decision: 02/02/2010, Positive, Last updated: 23/03/2015 -
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Opinion/decision on a Paediatric investigation plan (PIP): Sirturo, bedaquiline fumarate
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Infectious diseases
PIP number: EMEA-000912-PIP01-10-M04, Route(s) of administration: Oral use, Pharmaceutical form(s): Tablet, Granules
Decision date: 19/12/2018, Last updated: 18/02/2019, 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: Immunology-Rheumatology-Transplantation
PIP number: EMEA-000876-PIP05-15-M03, Route(s) of administration: Intravenous use, Pharmaceutical form(s): Concentrate for solution for infusion
Decision date: 19/07/2018, Last updated: 22/11/2018, Compliance check: X -
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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 first decision: 26/08/2005, Positive, Last updated: 01/04/2014 -
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Opinion/decision on a Paediatric investigation plan (PIP): Soliris, Eculizumab
Decision type: P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
Therapeutic area: Immunology-Rheumatology-Transplantation
PIP number: EMEA-000876-PIP06-15, Route(s) of administration: Intravenous use, Pharmaceutical form(s): Concentrate for solution for infusion
Decision date: 28/07/2016, Last updated: 20/04/2018, Compliance check: X -
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Orphan designation: carfilzomib for: Treatment of multiple myeloma
Date of first decision: 03/06/2008, Positive, Last updated: 02/12/2015 -
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Opinion/decision on a Paediatric investigation plan (PIP): Natpar, Recombinant parathyroid hormone
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Endocrinology, -gynacology-fertility-metabolism
PIP number: EMEA-001526-PIP01-13-M03, Route(s) of administration: Subcutaneous use, Pharmaceutical form(s): Powder and solvent for solution for injection
Decision date: 17/04/2019, Last updated: 24/06/2019, Compliance check: X -
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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 first decision: 09/10/2015, Positive, Last updated: 28/09/2017 -
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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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Opinion/decision on a Paediatric investigation plan (PIP): Lartruvo, Olaratumab
Decision type: W: decision granting a waiver in all age groups for all conditions or indications
Therapeutic area: Oncology
PIP number: EMEA-001760-PIP01-15-M03, Route(s) of administration: Intravenous use, Pharmaceutical form(s): Concentrate for solution for infusion
Decision date: 22/03/2019, Last updated: 24/06/2019, Compliance check: X -
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Orphan designation: Recombinant IgG degrading enzyme of Streptococcus pyogenes (imlifidase) for: Prevention of graft rejection following solid organ transplantation
Date of first decision: 12/01/2017, Positive, Last updated: 28/08/2020 -
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Opinion/decision on a Paediatric investigation plan (PIP): Tagrisso, Osimertinib (as mesylate)
Decision type: W: decision granting a waiver in all age groups for all conditions or indications
Therapeutic area: Oncology
PIP number: EMEA-002125-PIP01-17, Route(s) of administration: Oral use, Pharmaceutical form(s): Film-coated tablet
Decision date: 09/08/2017, Last updated: 28/09/2017, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Sovaldi, Sofosbuvir
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Infectious diseases
PIP number: EMEA-001276-PIP01-12-M02, Route(s) of administration: Oral use, Pharmaceutical form(s): Film-coated tablet, Age-appropriate oral solid dosage form, Other
Decision date: 15/06/2018, Last updated: 28/11/2018, Compliance check: X -
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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 -
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Opinion/decision on a Paediatric investigation plan (PIP): Isentress, Raltegravir
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Infectious diseases
PIP number: EMEA-000279-PIP01-08-M05, Route(s) of administration: Oral use, Pharmaceutical form(s): Film-coated tablet, Chewable tablet, Granules for oral suspension
Decision date: 15/06/2016, Last updated: 02/03/2018, Compliance check: V, 19/06/2017 -
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Opinion/decision on a Paediatric investigation plan (PIP): Prepandrix, Purified antigen fractions of inactivated split virion Influenza A/Vietnam/1194/2004 (H5N1) like strain used (NIBRG-14), Purified antigen fractions of inactivated split virion Influenza A/Indonesia/05/2005 (H5N1) like strain used (PR8-IBCDC-RG2)
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Vaccines
PIP number: EMEA-000160-PIP01-07-M05, Route(s) of administration: Intramuscular use, Pharmaceutical form(s): Emulsion and suspension for emulsion for injection
Decision date: 14/08/2019, Last updated: 20/11/2019, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Darzalex, Daratumumab
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Oncology
PIP number: EMEA-002152-PIP02-17-M01, Route(s) of administration: Intravenous use, Subcutaneous use, Pharmaceutical form(s): Solution for injection, Concentrate for solution for infusion
Decision date: 12/06/2019, Last updated: 08/11/2019, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Viekirax, Ombitasvir, paritaprevir, ritonavir
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Infectious diseases
PIP number: EMEA-001440-PIP01-13-M02, Route(s) of administration: Oral use, Pharmaceutical form(s): Tablet, Age-appropriate oral solid dosage form
Decision date: 15/06/2018, Last updated: 22/11/2018, Compliance check: X -
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Orphan designation: budesonide for: Treatment of eosinophilic oesophagitis
Date of first decision: 05/08/2013, Positive, Last updated: 18/01/2018 -
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Orphan designation: Cyclopropane-1,1-dicarboxylic acid [4-(6,7-dimethoxy-quinolin-4-yloxy)-phenyl]-amide (4-fluoro-phenyl)-amide, (L)-malate salt (cabozantinib) for: Treatment of medullary thyroid carcinoma
Date of first decision: 06/02/2009, Positive, Last updated: 12/05/2014 -
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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