Table 2 PIP details of ODs that are authorised for use in children
Medicine name (active substance) | Paediatric use | Paediatric investigation plan | ||||
|---|---|---|---|---|---|---|
Potential paediatric* | On label† | Decision‡ | Condition and age covered by waiver | Ground for waiver | Expected date of completion § | |
Elaprase (idursulfase) | Yes | All | PW | Mucopolysaccharidosis II (Hunter syndrome) (Girls birth to < 18 y) | Condition does not occur in the specified paediatric subset | December 2015 |
Exjade¶ (deferasirox) | Yes | 2 | PW | Chronic iron overload requiring chelation therapy (birth to < 2 years) | No significant therapeutic benefit | June 2015 |
Glivec* (imatinib) | Yes | 1 | PW | Pulmonary arterial hypertension (PAH) (birth to <6 months) | Likely ineffective | May 2013: PIP completed |
Philadelphia chromosome (BCR-ABL translocation) - positive chronic myeloid leukaemia (birth to <18 years) | No significant therapeutic benefit | |||||
Treatment of Philadelphia chromosome (BCR-ABL translocation) - positive acute lymphoblastic leukaemia (birth to < 1 year). | Condition does not occur in the specified paediatric subset | |||||
Ilaris (canakinumab) | Yes | 2 | PW | Juvenile idiopathic arthritis (birth to < 24 months) | Condition does not occur in the specified paediatric subset and no significant therapeutic benefit | June 2015 |
Cryopyrin Associated Periodic Syndromes (CAPS) including: FCAS, FCU, MWS, NOMID and CINCA* (birth to < 28 days) | No significant therapeutic benefit | |||||
Inovelon (rufinamide) | Yes | 4 | PW | Lennox-Gastaut syndrome (birth to < 12 months and from 4 to <18 years) | Condition does not occur in the specified paediatric subset and no significant therapeutic benefit | September 2017 |
Kuvan (sapropterin dihydrochloride) | Yes | 4 | PW | Hyperphenylalaninemia (4 to < 18 years) | No significant therapeutic benefit | January 2014 |
Mozobil (Plerixafor) | Yes | All | PW | Myelosuppression caused by chemotherapy to treat malignant disorders, which requires an autologous haematopoietic stem cell transplant (birth to < 12 months ) | No significant therapeutic benefit | June 2017 |
Novothirtheen (catridecacog) | Yes | 6 | PW | Prevention of bleeding during surgical interventions in congenital factor XIII A-subunit deficiency and treatment of bleeding in congenital factor XIII A-subunit deficiency (birth to <18 years) | Condition does not occur in the specified paediatric subset | December 2015 |
For the prevention of bleeding in congenital factor XIII A-subunit deficiency (birth to <1 year) | No significant therapeutic benefit | |||||
Tobi Podhaler (tobramycin) | Yes | 6 | PW | Pseudomonas aeruginosa pulmonary infection/colonisation in patients with cystic fibrosis (birth to < 3 months) | Likely unsafe and no significant therapeutic benefit | September 2015 |
Tracleer¶ (bosentan monohydrate) | Yes | 3 | PW | Systemic sclerosis and of interstitial pulmonary (birth to < 18 years) | Condition does not occur in the specified paediatric subset | December 2013 |
Pulmonary arterial hypertension (PAH) (from 28 days to < 3 months and from 12 – 18 years) | No significant therapeutic benefit | |||||
Votubia (everolimus) | Yes | 3 | PW | Angiomyolipoma (birth to < 18 years) | Condition does not occur in the specified paediatric subset | March 2020 |
Subependymal giant cell astrocytoma and tuberous Sclerosis Complex (NA) | NA | |||||
Vpriv (velaglucerase alfa) | Yes | 3 | PW | Gaucher Disease, Type 2 (birth to < 18 years) | Likely ineffective | July 2015 |
Gaucher Disease, types 1 and 3 (from birth to < 24 months) | No significant therapeutic benefit | |||||
Xagrid¶ (anagrelide) | Yes | All | PW | Essential Thrombocythaemiaa (birth to < 6 years) | Condition does not occur in the specified paediatric subset | March 2013 |
Kalydeco (ivacaftor) | Yes | 6 | FP | NA | NA | December 2016 |
Orfadin¶ | Yes | All | FP | NA | NA | May 2013 |
Revatio¶ | Yes | 1 | FP | NA | NA | July 2014 |