Displaying 361 - 380 of 773
PIPS Search Results
PIP Number Active Substance Conditions / Indications Invented Name Therapeutic area Sort descending Decision Type Compliance Check Compliance outcome Date Published
MHRA-100187-PIP01-21-M01 (update)
  • VONICOG ALFA
  • Treatment of von Willebrand Disease
  • Haematology-Hemostaseology
PM: decision on the application for modification of an agreed paediatric investigation plan. No 07/12/2022
MHRA-100302-PIP01-21-M01 (update)
  • CRIZANLIZUMAB
  • Treatment of sickle cell disease
  • Adakveo® 10 mg/ml concentrate for solution for infusion
  • Haematology-Hemostaseology
PM: decision on the application for modification of an agreed paediatric investigation plan. No 07/12/2022
MHRA-100412-PIP01-22-M02 (update)
  • vadadustat
  • Treatment of anaemia due to chronic disorders
  • VAFSEO
  • VAFSEO
  • Haematology-Hemostaseology
PM: decision on the application for modification of an agreed paediatric investigation plan. No 07/12/2022
MHRA-100465-PIP01-22-M01 (update)
  • efanesoctocog alfa
  • Treatment of congenital haemophilia A
  • Haematology-Hemostaseology
PM: decision on the application for modification of an agreed paediatric investigation plan. No 07/12/2022
MHRA-100608-PIP01-22-M01 (update)
  • mitapivat
  • Treatment of pyruvate kinase deficiency
  • PYRUKYND
  • PYRUKYND
  • Haematology-Hemostaseology
PM: decision on the application for modification of an agreed paediatric investigation plan. Yes Partial Compliance Check Granted 07/12/2022
MHRA-100695-PIP01-22-M01 (update)
  • Etranacogene dezaparvovec
  • Treatment of haemophilia B
  • Hemgenix
  • Haematology-Hemostaseology
PM: decision on the application for modification of an agreed paediatric investigation plan. No 02/02/2023
MHRA-100406-PIP01-21-M01 (update)
  • Marstacimab
  • Treatment of congenital haemophilia A
  • Treatment of congenital haemophilia B
  • Haematology-Hemostaseology
PM: decision on the application for modification of an agreed paediatric investigation plan. No 02/02/2023
MHRA-100695-PIP01-22-M02 (update)
  • Etranacogene dezaparvovec
  • Treatment of haemophilia B
  • Hemgenix
  • Haematology-Hemostaseology
PM: decision on the application for modification of an agreed paediatric investigation plan. No 02/02/2023
MHRA-100169-PIP01-21-M01 (update)
  • BETIBEGLOGENE AUTOTEMCEL
  • Treatment of beta-thalassaemia
  • zynteglo
  • Haematology-Hemostaseology
PM: decision on the application for modification of an agreed paediatric investigation plan. No 02/02/2023
MHRA-100499-PIP01-22-M01 (update)
  • Giroctocogene Fitelparvovec
  • Treatment of haemophilia A
  • Haematology-Hemostaseology
PM: decision on the application for modification of an agreed paediatric investigation plan. No 02/02/2023
MHRA-100451-PIP01-22-M01 (update)
  • fidanacogene elaparvovec
  • Treatment of congenital factor IX deficiency (haemophilia B)
  • Haematology-Hemostaseology
PM: decision on the application for modification of an agreed paediatric investigation plan. No 02/02/2023
MHRA-100451-PIP01-22-M02 (update)
  • fidanacogene elaparvovec
  • Treatment of congenital factor IX deficiency (haemophilia B)
  • Haematology-Hemostaseology
PM: decision on the application for modification of an agreed paediatric investigation plan. No 02/02/2023
MHRA-100559-PIP01-22
  • mitapivat
  • Treatment of thalassaemia
  • PYRUKYND
  • PYRUKYND
  • Haematology-Hemostaseology
P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 25/05/2023
MHRA-100635-PIP01-22
  • CEDAZURIDINE
  • DECITABINE
  • Treatment of acute myeloid leukaemia
  • INQOVI
  • INQOVI
  • Inqovi 35/100
  • INAQOVI
  • Haematology-Hemostaseology
  • Oncology
P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) No 25/05/2023
MHRA-100213-PIP01-21-M03 (update)
  • RAVULIZUMAB
  • Treatment of paroxysmal nocturnal haemoglobinuria (PNH)
  • Ultomiris
  • Haematology-Hemostaseology
PM: decision on the application for modification of an agreed paediatric investigation plan. Yes 25/05/2023
MHRA-100214-PIP01-21-M03 (update)
  • RAVULIZUMAB
  • atypical haemolytic uremic syndrome (aHUS)
  • Haematology-Hemostaseology
PM: decision on the application for modification of an agreed paediatric investigation plan. Yes 25/05/2023
MHRA-100816-PIP01-22-M01 (update)
  • crovalimab
  • Treatment of paroxysmal nocturnal haemoglobinuria
  • Treatment of atypical haemolytic uremic syndrome
  • Haematology-Hemostaseology
PM: decision on the application for modification of an agreed paediatric investigation plan. Yes 25/05/2023
MHRA-100544-PIP01-22-M01 (update)
  • sebetralstat
  • Treatment of hereditary angioedema
  • Haematology-Hemostaseology
PM: decision on the application for modification of an agreed paediatric investigation plan. No 30/06/2023
MHRA-100802-PIP01-22-M01 (update)
  • roxadustat
  • Treatment of anaemia due to chronic disorders
  • EVRENZO
  • EVRENZO
  • EVRENZO
  • EVRENZO
  • EVRENZO
  • EVRENZO
  • EVRENZO
  • EVRENZO
  • Haematology-Hemostaseology
PM: decision on the application for modification of an agreed paediatric investigation plan. No 30/06/2023
MHRA-100614-PIP01-22-M01 (update)
  • VONICOG ALFA
  • Treatment of von Willebrand Disease
  • veyvondi
  • veyvondi
  • Vonvendi
  • veyvondi
  • Vonvendi
  • veyvondi
  • Vonvendi
  • veyvondi
  • Haematology-Hemostaseology
PM: decision on the application for modification of an agreed paediatric investigation plan. No 30/06/2023