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Table 2 PIP details of ODs that are authorised for use in children

From: The influence of the European paediatric regulation on marketing authorisation of orphan drugs for 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
  1. *Intended for the paediatric population at time of ODD (yes/no).
  2. †Minimum age (in years) on SmPC at time of MA. All: age range not specified and/ or no age contraindication.
  3. ‡PIP decision granted by EMA: PW: partial waiver, FP: Full investigation plan, for the entire paediatric population. NA: Not applicable.
  4. §Expected date of PIP completion for the remaining population.
  5. ¶Product was authorised before 2007 however the MAH applied for or had the intention to apply for an extension of the authorised indication. Consequently, pursuant to Article 8 of Regulation (EC) No 1901/2006, the MAH submitted a PIP.
  6. FCAS: Familial Cold Autoinflammatory Syndrome; FCU: Familial Cold Urticaria; MWS: Muckle-Wells Syndrome; NOMID: Neonatal-Onset Multisystem Inflammatory Disease; CINCA: Chronic Infantile Neurological, Cutaneous, Articular Syndrome.
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172