639 results
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Orphan designation: Mixture of two adeno-associated viral vectors of serotye 8 containing the 5'-half sequence of human ABCA4 gene and the 3'-half sequence of human ABCA4 gene for: Treatment of Stargardt's disease
Date of designation: 04/07/2014, Positive, Last updated: 07/11/20225'-half sequence of human ABCA4 gene and the 3'-half sequence … 3'-half sequence of human ABCA4 gene Overview On 4 July 2014 … 5'-half sequence of human ABCA4 gene and the 3'-half sequence … -
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Orphan designation: Lentiviral vector containing the human ABCA4 gene for: Treatment of Stargardt's disease (updated)
Date of designation: 02/02/2010, Positive, Last updated: 13/03/2023vector containing the human ABCA4 gene Overview On 2 February … vector containing the human ABCA4 gene for the treatment of … abnormalities in a gene called ABCA4. The … -
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Opinion/decision on a Paediatric investigation plan (PIP): Aciclovir
Decision type: W: decision granting a waiver in all age groups for all conditions or indications
Therapeutic area: Infectious diseases
PIP number: EMEA-001066-PIP02-11-M03, Route(s) of administration: Gingival use, Pharmaceutical form(s): Muco-adhesive buccal tablet
Decision date: 04/12/2019, Last updated: 01/04/2020, Compliance check: XSitavig and associated names Aciclovir Infectious diseasesP/0412/2019EMEA-001066-PIP02-11-M03 … paediatric investigation plan for aciclovir (Sitavig and associated names … Aciclovir … -
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Opinion/decision on a Paediatric investigation plan (PIP): Lentiviral vector containing the human ABCA4 gene
Decision type: P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
Therapeutic area: Ophthalmology
PIP number: EMEA-002407-PIP01-18, Route(s) of administration: Intraocular use, Pharmaceutical form(s): Solution for injection
Decision date: 14/06/2019, Last updated: 07/11/2019, Compliance check: Xvector containing the human ABCA4 gene OphthalmologyP/0213/2019EMEA-002407-PIP01-18 … vector containing the human ABCA4 gene (SAR422459) (EMEA-002407-PIP01-18 … vector containing the human ABCA4 gene … -
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Opinion/decision on a Paediatric investigation plan (PIP): Aciclovir
Decision type: RW: decision refers to a refusal on a request for waiver in all age groups for the listed condition(s)
Therapeutic area: Infectious diseases
PIP number: EMEA-001066-PIP01-10, Route(s) of administration: Gingival use, Pharmaceutical form(s): Muco-adhesive buccal tablet
Decision date: 06/04/2011, Last updated: 12/05/2011, Compliance check: XKey facts Aciclovir Infectious diseasesP/83/2011EMEA-001066-PIP01-10 … product specific waiver for aciclovir (EMEA-001066-PIP01-10) PDF … product specific waiver for aciclovir (EMEA-001066-PIP01-10) in … -
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Orphan designation: Adeno-associated viral vector serotype 5 containing the human ABCA4 gene for: Treatment of Stargardt's disease
Date of designation: 07/02/2009, Withdrawn, Last updated: 16/10/2014serotype 5 containing the human ABCA4 gene Overview Please note … serotype 5 containing the human ABCA4 gene for the treatment of … abnormalities in a gene called ABCA4. The … -
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Orphan designation: Allogeneic skin-derived ABCB5-positive mesenchymal stem cells for: Treatment of epidermolysis bullosa
Date of designation: 29/05/2019, Positive, Last updated: 09/08/2019Allogeneic skin-derived ABCB5-positive mesenchymal stem cells Overview … for allogeneic skin-derived ABCB5-positive mesenchymal stem cells (also … of allogeneic skin-derived ABCB5-positive mesenchymal stem cells have … -
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Orphan designation: Adeno-associated viral vector serotype 9 containing the human ABCD1 gene for: Treatment of adrenoleukodystrophy
Date of designation: 11/11/2022, Positive, Last updated: 25/01/2023serotype 9 containing the human ABCD1 gene Overview This medicine … serotype 9 containing the human ABCD1 gene … -
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Opinion/decision on a Paediatric investigation plan (PIP): Aciclovir, hydrocortisone
Decision type: W: decision granting a waiver in all age groups for all conditions or indications
Therapeutic area: Infectious diseases
PIP number: EMEA-000110-PIP01-07, Route(s) of administration: Topical use, Pharmaceutical form(s): Cream
Decision date: 15/08/2008, Last updated: 18/09/2008, Compliance check: XKey facts Aciclovir hydrocortisone Infectious … Paediatric Investigation Plan for aciclovir, hydrocortisone, (EMEA-000110-PIP01-07 … Word - Decision and Opinion aciclovir - hydrocortisone 000110-PIP… … -
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Orphan designation: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA (elivaldogene autotemcel) for: Treatment of adrenoleukodystrophy
Date of designation: 06/06/2012, Withdrawn, Last updated: 04/04/2022Lenti-D encoding the human ABCD1 cDNA (elivaldogene autotemcel … Lenti-D encoding the human ABCD1 cDNA was authorised in the … Lenti-D encoding the human ABCD1 cDNA for the treatment of … -
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Orphan designation: Combination of two adeno-associated viral vectors of serotype 8 containing the 5'- and the 3'- half coding sequences of human ABCA4 fused to inteins for: Treatment of Stargardt's disease
Date of designation: 17/10/2019, Positive, Last updated: 07/11/2022coding sequences of human ABCA4 fused to inteins Overview … coding sequences of human ABCA4 fused to inteins for the … abnormalities in a gene called ABCA4. The … -
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Opinion/decision on a Paediatric investigation plan (PIP): Allogeneic skin-derived ATP-binding cassette, sub-family B, member 5 (ABCB5)-positive mesenchymal stem cells (allo-APZ2-EB)
Decision type: P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
Therapeutic area: Dermatology
PIP number: EMEA-002875-PIP01-20, Route(s) of administration: Intravenous use, Pharmaceutical form(s): Suspension for injection
Decision date: 09/07/2021, Last updated: 19/07/2022, Compliance check: Xcassette, sub-family B, member 5 (ABCB5)-positive mesenchymal stem … cassette, sub-family B, member 5 (ABCB5)-positive mesenchymal stem … sub-family B, member 5 (ABCB5)-positive mesenchymal stem … -
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Opinion/decision on a Paediatric investigation plan (PIP): autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ATP-binding cassette, sub-family D (ALD), member 1 (ABCD1) from cDNA
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Neurology
PIP number: EMEA-001244-PIP01-11-M02, Route(s) of administration: Intravenous use, Pharmaceutical form(s): Dispersion for infusion
Decision date: 12/09/2018, Last updated: 16/09/2021, Compliance check: V, 04/09/2020sub-family D (ALD), member 1 (ABCD1) from cDNA NeurologyP/0290/2018EMEA-001244-PIP01-11-M02 … sub-family D (ALD), member 1 (ABCD1) from cDNA … -
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Referral: GVK Biosciences
esomeprazole magnesium, candesartan cilexetil, nebivolol, desloratadine, donepezil, repaglinide, candesartan, esomeprazole, alendronic acid, ropinirole, cefpodoxime, quetiapine, trimetazidine, bosentan, tramadol, paracetamol, escitalopram, trimetazidine hydrochloride, entacapone, valsartan, hydrochlorothiazide, donepezil hydrochloride, ropinirole hydrochloride, clopidogrel hydrochloride, escitalopram oxalate, metformin hydrochloride, trimetazidine dihydrochloride, rizatriptan benzoate, metformin, fluconazole, eletriptan, rizatriptan, ebastine, ibuprofen, desmopressin, aciclovir, levetiracetam, irbesartan, venlafaxine, ciprofloxacin, cefpodoxime proxetil, tacrolimus, fexofenadine hydrochloride, phenoxymethylpenicillin, amlodipine besilate, metoclopramide, clopidogrel, levodopa, carbidopa, entacapone, atorvastatin, telmisartan, clindamycin hydrochloride, metoclopramide hydrochloride, clonazepam, clobazam, dexamethasone, thiamine hydrochloride, tramadol hydrochloride, pioglitazone, dipyridamole, fexofenadine, amlodipine, pantoprazole, hydrocortisone, Article 31 referrals
Status: European Commission final decision, opinion/position date: 21/05/2015, EC decision date: 16/07/2015, Last updated: 04/08/2015benzoate metformin fluconazole eletriptan rizatriptan ebastine ibuprofen desmopressin aciclovir levetiracetam irbesartan venlafaxine ciprofloxacin cefpodoxime … -
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Human medicine European public assessment report (EPAR): Skysona
elivaldogene autotemcel, Adrenoleukodystrophy
Date of authorisation: 16/07/2021,
, Withdrawn, Last updated: 04/04/2022
18 years of age, with an ABCD1 genetic mutation, and for … change (mutation) in the ABCD1 gene. The mutation prevents … -
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Orphan designation: Recombinant human apolipoprotein A-I in a complex with phospholipids for: Treatment of ATP-binding cassette transporter A1 deficiency
Date of designation: 22/08/2014, Positive,cassette transporter A1 (ABCA1) is a protein that regulates … the body. In patients with ABCA1 deficiency, the ABCA1 protein is not produced in … mutation (defect) in their ABCA1 genes, and so cholesterol … -
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Orphan designation: Allogeneic ABCB5-positive limbal stem cells for: Treatment of limbal stem cell deficiency
Date of designation: 14/12/2018, Positive, Last updated: 11/03/2019Allogeneic ABCB5-positive limbal stem cells Overview … KG, Germany, for allogeneic ABCB5-positive limbal stem cells (also known … -
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Referral: Valtrex
valaciclovir, associated names: Zelitrex, Talavir, Valaciclovir Allen, Valherpes, Article 30 referrals
Status: European Commission final decision, opinion/position date: 22/04/2010, EC decision date: 13/07/2010, Last updated: 25/03/2014body into two substances, aciclovir and valine (an amino-acid Aciclovir is an antiviral that works … body into two substances, aciclovir and valine (an amino-acid … and valine (an amino-acid). Aciclovir is an antiviral that works … -
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Orphan designation: Ecothiopate iodide for: Treatment of Stargardt's disease
Date of designation: 24/04/2015, Withdrawn, Last updated: 10/12/2020abnormalities in a gene called ABCA4 . The ABCA4 gene is responsible for the … production of a protein called ABCR that regulates the transport … with Stargardt's disease, ABCR does not work properly. This … -
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Orphan designation: Soraprazan for: Treatment of Stargardt's disease
Date of designation: 13/11/2013, Positive, Last updated: 02/12/2013abnormalities in a gene called ABCA4. The ABCA4 gene is responsible for the … production of a protein called ABCR that regulates the transport … with Stargardt's disease, ABCR does not work properly. This … -
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Orphan designation: Emixustat hydrochloride for: Treatment of Stargardt's disease
Date of designation: 29/05/2019, Positive, Last updated: 09/08/2019abnormalities in a gene called ABCA4. The ABCA4 gene is responsible for the … production of a protein called ABCR that regulates the transport … with Stargardt’s disease, ABCR does not work properly. As … -
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Orphan designation: Ramiprilat for: Treatment of Stargardt's disease
Date of designation: 12/03/2013, Positive, Last updated: 17/01/2023abnormalities in a gene called ABCA4. The ABCA4 gene is responsible for the … production of a protein called ABCR that regulates the transport … with Stargardt's disease, ABCR does not work properly. This … -
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Orphan designation: 1-(3-{4-[3,4-difluoro-2-(trifluoromethyl)phenyl]piperidine-1-carbonyl}-1H,4H,5H,6H,7H-pyrazolo[3,4- c]pyridin-6-yl)ethan-1-one (Tinlarebant) for: Treatment of Stargardt's disease
Date of designation: 25/05/2018, Positive, Last updated: 01/12/2022abnormalities in a gene called ABCA4 . The ABCA4 gene is responsible for the … production of a protein called ABCR that regulates the transport … with Stargardt's disease, ABCR does not work properly. This … -
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Orphan designation: 3-(3-(3,5-bis(trifluoromethyl)phenyl)-1h-pyrazol-1-yl)propanoic acid for: Treatment of Stargardt's disease
Date of designation: 24/04/2019, Positive, Last updated: 24/05/2022abnormalities in a gene called ABCA4. The ABCA4 gene is responsible for the … production of a protein called ABCR that regulates the transport … with Stargardt’s disease, ABCR does not work properly. As … -
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Orphan designation: Etidronate disodium for: Treatment of pseudoxanthoma elasticum
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023medicine for the treatment of ABCC6 deficiency in the European … medicine is expected to work The ABCC6 gene is involved in transporting … inherited mutations in the ABCC6 gene, ATP is insufficiently …