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PIPS Search Results
PIP Number Active Substance Conditions / Indications Invented Name Therapeutic area Decision Type Compliance Check Compliance outcome Date Published
MHRA-100100-PIP01-21
  • Human Immunoglobulin G1 Constant Region – Human Ectodysplasin-A1 Receptor-binding Domain Fusion Protein
  • Treatment of X-linked hypohidrotic ectodermal dysplasia (XLHED)
  • Dermatology
 P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 29/07/2022
MHRA-100109-PIP01-21
  • Islatravir
  • Prevention of human immunodeficiency virus (HIV-1) infection
  • Infectious diseases
 P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 29/07/2022
MHRA-100165-PIP01-21
  • PF-06865571
  • Treatment of non-alcoholic steatohepatitis
  • PF-06865571
  • Gastroenterology-Hepatology
 P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 29/07/2022
MHRA-100199-PIP01-21
  • efgartigimod alfa
  • Treatment of Pemphigus
  • Dermatology
 W: decision granting a waiver in all age groups for the listed condition(s). No 29/07/2022
MHRA-100265-PIP01-21
  • BARICITINIB
  • Treatment of alopecia areata
  • Olumiant
  • Olumiant
  • Immunology -Rheumatology-Transplantation
 P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 29/07/2022
MHRA-100293-PIP01-21
  • lenzilumab
  • Treatment of coronavirus disease 2019 (COVID-19)
  • Not available at present
  • Immunology -Rheumatology-Transplantation
 P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 29/07/2022
MHRA-100396-PIP01-21
  • sugemalimab
  • Treatment of non-small cell lung cancer (NSCLC)
  • Oncology
 W: decision granting a waiver in all age groups for the listed condition(s). No 29/07/2022
MHRA-100224-PIP01-21
  • Etrasimod L-arginine
  • Treatment of ulcerative colitis
  • Not available at present
  • Gastroenterology-Hepatology
 PM: decision on the application for modification of an agreed paediatric investigation plan. No 29/07/2022
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-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-100312-PIP01-21
  • Ritlectinib
  • Treatment of alopecia areata
  • Dermatology
 P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) Yes Partial Compliance Check Granted 29/07/2022
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
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