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PIP Number Active Substance Conditions / Indications Invented Name Therapeutic area Decision Type Compliance Check Compliance outcome Date Published
MHRA-100503-PIP01-22
  • efavaleukin alfa
  • Treatment of ulcerative colitis
  • Not available at present
  • Other: Inflammatory Bowel Disease
 P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 25/05/2023
MHRA-100811-PIP01-22
  • Vamorolone
  • Treatment of Duchenne muscular dystrophy
  • AGAMREE
  • Other: Neuromuscular Diseases
 PM: decision on the application for modification of an agreed paediatric investigation plan. No 25/05/2023
MHRA-100811-PIP01-22-M01 (update)
  • Vamorolone
  • Treatment of Duchenne muscular dystrophy
  • Other: Neuromuscular Diseases
 PM: decision on the application for modification of an agreed paediatric investigation plan. Yes Partial Compliance Check Granted 25/05/2023
MHRA-100274-PIP01-21-M02 (update)
  • ELIGLUSTAT
  • Treatment of Gaucher Disease Type 2
  • Treatment of Gaucher Disease Type 1 and Type 3
  • CERDELGA
  • Other: Lysosomal Storage Disorder
 PM: decision on the application for modification of an agreed paediatric investigation plan. No 25/05/2023
MHRA-100274-PIP01-21-M01 (update)
  • ELIGLUSTAT
  • Treatment of Gaucher Disease type 2
  • Treatment of Gaucher Disease Type 1 and Type 3
  • CERDELGA
  • Other: Lysosomal Storage Disorder
 PM: decision on the application for modification of an agreed paediatric investigation plan. No 25/05/2023
MHRA-100630-PIP01-22-M01 (update)
  • PATIROMER SORBITEX CALCIUM
  • Treatment of hyperkalaemia
  • Veltassa
  • Other: Nephrology/Metabolism
 PM: decision on the application for modification of an agreed paediatric investigation plan. Yes Partial Compliance Check Granted 25/05/2023
MHRA-100532-PIP01-22-M02 (update)
  • BUROSUMAB
  • Treatment of X-linked Hypophosphataemia
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Crysvita
  • Other: musculoskeletal disorder; osteology
 PM: decision on the application for modification of an agreed paediatric investigation plan. No 25/05/2023
MHRA-100681-PIP01-22
  • Lomitapide
  • Treatment of (heterozygous or homozygous) familial hypercholesterolaemia
  • Lojuxta hard capsule
  • Other: Hypercholesterolaemia (HoFH)
 PM: decision on the application for modification of an agreed paediatric investigation plan. No 30/06/2023
MHRA-100727-PIP01-22-M01 (update)
  • ALPELISIB
  • Treatment of PIK3CA related overgrowth spectrum
  • Vijoice
  • Piqray
  • Vijoice
  • Other: Congenital, Hereditary, Neonatal Disease and Abnormalities
PM: decision on the application for modification of an agreed paediatric investigation plan. No 30/06/2023
MHRA-100805-PIP01-22-M01 (update)
  • ATIDARSAGENE AUTOTEMCEL
  • Treatment of metachromatic leukodystrophy (MLD)
  • Libmeldy
  • Libmeldy
  • Libmeldy
  • Other: Lysosomal storage Disorder
 PM: decision on the application for modification of an agreed paediatric investigation plan. No 30/06/2023
MHRA-100820-PIP01-22
  • Tirzepatide
  • Treatment of obesity
  • Mounjaro
  • Other: Obesity, Overweight
 P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 24/08/2023
MHRA-100750-PIP01-22-M01 (update)
  • setrusumab
  • Treatment of osteogenesis imperfecta
  • Other: Skeletal
 PM: decision on the application for modification of an agreed paediatric investigation plan. No 24/08/2023
MHRA-100844-PIP01-23-M01 (update)
  • IVACAFTOR
  • Treatment of cystic fibrosis.
  • Kalydeco
  • Kalydeco
  • Kalydeco
  • Kalydeco
  • Kalydeco
  • Kalydeco
  • Kalydeco
  • Other: Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 PM: decision on the application for modification of an agreed paediatric investigation plan. Yes Full Compliance Check Granted 24/08/2023
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-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
MHRA-100023-PIP01-21-M01 (update)
  • bimekizumab
  • Treatment of psoriasis
  • Bimzelx
  • Dermatology
 PM: decision on the application for modification of an agreed paediatric investigation plan. No 03/11/2021
MHRA-100090-PIP01-21-M01 (update)
  • DUPILUMAB
  • Treatment of atopic dermatitis
  • Dupixent
  • Dupixent
  • Dermatology
 PM: decision on the application for modification of an agreed paediatric investigation plan. No 18/03/2022
MHRA-100276-PIP01-21-M01 (update)
  • BRODALUMAB
  • Treatment of psoriasis
  • brodalumab
  • Lumicef
  • Siliq
  • Kyntheum
  • Dermatology
 PM: decision on the application for modification of an agreed paediatric investigation plan. No 04/05/2022
MHRA-100216-PIP01-21-M01 (update)
  • TRALOKINUMAB
  • Treatment of atopic dermatitis
  • Adtralza
  • Adtralaza
  • Adtralza
  • Dermatology
 PM: decision on the application for modification of an agreed paediatric investigation plan. No 17/05/2022
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