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Displaying 201 - 220 of 669
PIPS Search Results
PIP Number Active Substance Sort descending Conditions / Indications Invented Name Therapeutic area Decision Type Compliance Check Compliance outcome Date Published
MHRA-100244-PIP01-21
  • surufatinib
  • Treatment of malignant neoplasms
  • Malignant neoplasms of haematopoietic and lymphoid tissue
  • Sevsury
  • Sulanda
  • Oncology
P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 29/07/2022
MHRA-100270-PIP01-21
  • crinecerfont
  • Treatment of congenital adrenal hyperplasia
  • Endocrinology-Gynaecology-Fertility-metabolism
P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 29/07/2022
MHRA-100270-PIP01-21-M01 (update)
  • crinecerfont
  • Treatment of congenital adrenal hyperplasia
  • Endocrinology-Gynaecology-Fertility-metabolism
PM: decision on the application for modification of an agreed paediatric investigation plan. No 19/12/2023
MHRA-100266-PIP01-21
  • Autologous CD34+ hematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene
  • Treatment of sickle cell disease
  • Haematology-Hemostaseology
P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 29/07/2022
MHRA-100267-PIP01-21
  • Autologous CD34+ hematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene
  • Treatment of beta-thalassemia intermedia and major
  • Haematology-Hemostaseology
P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 29/07/2022
MHRA-100305-PIP01-21
  • Single chain urokinase plasminogen activator (scuPA)
  • Treatment of pleural effusion
  • Pneumology - Allergology
P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 29/07/2022
MHRA-100304-PIP01-21
  • rozibafusp alfa
  • Treatment of systemic lupus erythematosus (SLE)
  • Not available at present
  • Immunology -Rheumatology-Transplantation
  • Other: Inflammation-Rheumatology SLE
P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 29/07/2022
MHRA-100364-PIP01-21
  • Respiratory Syncytial Virus (RSV) PreF3 recombinant Fusion protein/AS01
  • Prevention of lower respiratory tract disease caused by respiratory syncytial virus
  • Not available at present
  • Vaccines
P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 29/07/2022
MHRA-100316-PIP01-21
  • latozinemab
  • Treatment of clinically symptomatic or pre-symptomatic frontotemporal dementia (FTD)
  • Neurology
W: decision granting a waiver in all age groups for the listed condition(s). No 29/07/2022
MHRA-100315-PIP01-21
  • Brensocatib
  • Treatment of non‐cystic fibrosis bronchiectasis
  • Pneumology - Allergology
P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 29/07/2022
MHRA-100330-PIP01-21
  • Autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) genetically modified with the lentiviral vector IDUA LVV, encoding for the human α-L-iduronidase (IDUA) gene (OTL-203)
  • Treatment of Mucopolysaccharidosis type I, Hurler syndrome (MPS-IH)
  • Other: Metabolic disorders
P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 29/07/2022
MHRA-100330-PIP01-21 -M01 (update)
  • Autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) genetically modified with the lentiviral vector IDUA LVV, encoding for the human α-L-iduronidase (IDUA) gene (OTL-203)
  • Treatment of Mucopolysaccharidosis type I, Hurler syndrome (MPS-IH)
  • Not available at present
  • Other: Metabolic disorders
PM: decision on the application for modification of an agreed paediatric investigation plan. No 24/08/2023
MHRA-100448-PIP01-22
  • RUXOLITINIB PHOSPHATE
  • Treatment of vitiligo
  • OPZELURA
  • Dermatology
P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 29/07/2022
MHRA-100037-PIP01-21-M01 (update)
  • ponesimod
  • Treatment of Multiple Sclerosis
  • Ponvory
  • Neurology
  • Other: Autoimmune disease/disfunction
PM: decision on the application for modification of an agreed paediatric investigation plan. No 29/07/2022
MHRA-100037-PIP01-21-M02 (update)
  • ponesimod
  • Treatment of Multiple Sclerosis
  • Ponvory
  • Neurology
  • Other: Autoimmune disease/dysfunction
PM: decision on the application for modification of an agreed paediatric investigation plan. No 30/04/2024
MHRA-100058-PIP01-21-M01 (update)
  • romosozumab
  • Treatment of osteoporosis
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • Endocrinology-Gynaecology-Fertility-metabolism
PM: decision on the application for modification of an agreed paediatric investigation plan. No 29/07/2022
MHRA-100222-PIP01-21-M01 (update)
  • romosozumab
  • Treatment of osteoporosis
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • Endocrinology-Gynaecology-Fertility-metabolism
PM: decision on the application for modification of an agreed paediatric investigation plan. No 29/07/2022
MHRA-100222-PIP01-21-M02 (update)
  • romosozumab
  • Treatment of osteoporosis
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • EVENITY
  • Endocrinology-Gynaecology-Fertility-metabolism
PM: decision on the application for modification of an agreed paediatric investigation plan. No 19/12/2023
MHRA-100130-PIP01-21-M01 (update)
  • BUPIVACAINE
  • Postsurgical analgesia
  • EXPAREL liposomal
  • EXPAREL
  • EXPAREL
  • EXPAREL
  • EXPAREL
  • EXPAREL
  • EXPAREL
  • Pain
PM: decision on the application for modification of an agreed paediatric investigation plan. No 29/07/2022
MHRA-100130-PIP01-21-M02 (update)
  • BUPIVACAINE
  • Postsurgical analgesia
  • EXPAREL liposomal
  • EXPAREL
  • EXPAREL
  • EXPAREL
  • EXPAREL
  • Pain
PM: decision on the application for modification of an agreed paediatric investigation plan. No 30/06/2023