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
|