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24 results
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Orphan designation: Autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti CD19 CD28/CD3-zeta chimeric antigen receptor and cultured (KTE-X19) for: Treatment of mantle cell lymphoma
Date of first decision: 13/11/2019, Positive, Last updated: 25/01/2021 -
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Orphan designation: Ex vivo fused normal allogeneic human myoblast with autologous human myoblast derived from Duchenne muscular dystrophy affected donor for: Treatment of Duchenne muscular dystrophy
Date of first decision: 19/11/2019, Positive, Last updated: 25/02/2019 -
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Orphan designation: (2S,3R,4R,5S)-2-(hydroxymethyl)-1-pentylpiperidine-3,4,5-triol for: Treatment of GM2 gangliosidosis
Date of first decision: 13/11/2019, Positive, Last updated: 12/02/2020 -
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Orphan designation: Exendin (9-39) for: Treatment of congenital hyperinsulinism
Date of first decision: 13/11/2019, Positive, Last updated: 13/02/2020 -
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Orphan designation: Chimeric fibril-reactive IgG1k monoclonal antibody 11-1F4 for: Treatment of AL amyloidosis
Date of first decision: 13/11/2019, Positive, Last updated: 13/02/2020 -
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Orphan designation: Replication-incompetent, non-integrating, herpes simplex virus 1 vector expressing the human transglutaminase-1 enzyme for: Treatment of autosomal recessive congenital ichthyosis
Date of first decision: 13/11/2019, Positive, Last updated: 13/02/2020 -
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Orphan designation: 4-((E)-(5-(2-(2-((S)-2-((S)-1-(L-threonyl-L-lysyl)pyrrolidine-2-carboxamido)-5-guanidinopentanamido)acetamido)-2-carboxyethyl)-2-hydroxyphenyl)diazenyl)phenyl (2-(trimethylammonio)ethyl) phosphate for: Treatment of non-infectious uveitis
Date of first decision: 13/11/2019, Positive, Last updated: 12/02/2020 -
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Orphan designation: Camsirubicin for: Treatment of soft tissue sarcoma
Date of first decision: 13/11/2019, Positive, Last updated: 13/02/2020 -
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Orphan designation: Ganaxolone for: Treatment of CDKL5 deficiency disorder
Date of first decision: 13/11/2019, Positive, Last updated: 13/02/2020 -
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Opinion/decision on a Paediatric investigation plan (PIP): Doptelet, avatrombopag maleate
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Haematology-Hemostaseology
PIP number: EMEA-001136-PIP01-11-M01, Route(s) of administration: Oral use, Pharmaceutical form(s): Film-coated tablet, Powder for oral suspension
Decision date: 22/11/2019, Last updated: 12/01/2021, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Sodium thiosulfate
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Oncology
PIP number: EMEA-002147-PIP02-17-M01, Route(s) of administration: Intravenous use, Pharmaceutical form(s): Solution for infusion
Decision date: 08/11/2019, Last updated: 30/03/2020, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Beclometasone dipropionate, formoterol fumarate dihydrate, glycopyrronium bromide (CHF 5993)
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Pneumology-allergology
PIP number: EMEA-001875-PIP02-18-M01, Route(s) of administration: Inhalation use, Pharmaceutical form(s): Inhalation powder, Pressurised inhalation, solution
Decision date: 07/11/2019, Last updated: 30/03/2020, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Dupixent, dupilumab
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Dermatology
PIP number: EMEA-001501-PIP01-13-M06, Route(s) of administration: Subcutaneous use, Pharmaceutical form(s): Solution for injection
Decision date: 22/11/2019, Last updated: 30/03/2020, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Prevymis, Letermovir
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Infectious diseases
PIP number: EMEA-001631-PIP01-14-M04, Route(s) of administration: Oral use, Intravenous use, Pharmaceutical form(s): Film-coated tablet, Granules, Concentrate for solution for infusion
Decision date: 04/11/2019, Last updated: 30/03/2020, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Daklinza, Daclatasvir
Decision type: W: decision granting a waiver in all age groups for all conditions or indications
Therapeutic area: Infectious diseases
PIP number: EMEA-001191-PIP01-11-M03, Route(s) of administration: Oral use, Pharmaceutical form(s): Chewable tablet, Film-coated tablet
Decision date: 08/11/2019, Last updated: 30/03/2020, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Latuda, Lurasidone hydrochloride
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Psychiatry
PIP number: EMEA-001230-PIP01-11-M05, Route(s) of administration: Oral use, Pharmaceutical form(s): Film-coated tablet
Decision date: 04/11/2019, Last updated: 30/03/2020, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Plegridy, peginterferon beta-1a
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Neurology
PIP number: EMEA-001129-PIP01-11-M04, Route(s) of administration: Subcutaneous use, Intramuscular use, Pharmaceutical form(s): Solution for injection
Decision date: 14/11/2019, Last updated: 27/03/2020, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Volibris, ambrisentan
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Cardiovascular diseases
PIP number: EMEA-000434-PIP01-08-M06, Route(s) of administration: Oral use, Pharmaceutical form(s): Film-coated tablet, Dispersible tablet
Decision date: 08/11/2019, Last updated: 27/03/2020, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Lubiprostone
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Gastroentology-Hepatology
PIP number: EMEA-000245-PIP01-08-M06, Route(s) of administration: Oral use, Pharmaceutical form(s): Capsule, soft
Decision date: 04/11/2019, Last updated: 27/03/2020, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Human normal immunoglobulin
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Immunology-Rheumatology-Transplantation
PIP number: EMEA-001853-PIP01-15-M02, Route(s) of administration: Subcutaneous use, Pharmaceutical form(s): Solution for injection
Decision date: 18/11/2019, Last updated: 18/11/2019, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Ganaxolone
Decision type: P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
Therapeutic area: Neurology
PIP number: EMEA-002341-PIP01-18, Route(s) of administration: Oral use, Pharmaceutical form(s): Oral suspension, Age-appropriate oral liquid dosage form
Decision date: 04/11/2019, Last updated: 27/03/2020, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Ilaris, Canakinumab
Decision type: W: decision granting a waiver in all age groups for all conditions or indications
Therapeutic area: Immunology-Rheumatology-Transplantation
PIP number: EMEA-000060-PIP07-19, Route(s) of administration: Subcutaneous use, Pharmaceutical form(s): Powder for solution for injection, Solution for injection
Decision date: 07/11/2019, Last updated: 27/03/2020, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Selpercatinib
Decision type: P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
Therapeutic area: Oncology
PIP number: EMEA-002544-PIP01-18, Route(s) of administration: Oral use, Pharmaceutical form(s): Capsule, hard, Age-appropriate oral solid dosage form
Decision date: 08/11/2019, Last updated: 27/03/2020, Compliance check: X -
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Opinion/decision on a Paediatric investigation plan (PIP): Remimazolam (as besilate)
Decision type: P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
Therapeutic area: Anaesthesiology
PIP number: EMEA-001880-PIP02-19, Route(s) of administration: Intravenous use, Intranasal use, Pharmaceutical form(s): Powder for solution for injection or infusion, Nasal powder
Decision date: 04/11/2019, Last updated: 27/03/2020, Compliance check: X