Table 1.
Pharmaceutics and licenced dose*
| Nonproprietary name | Trade name and maximum dose | Generic/biosimilar version approved | Licenced indication in children | Excipients of significance | Common side effects of active ingredient | Known side effects of excipient |
|---|---|---|---|---|---|---|
| Recombinant erythropoietin (Epoetin Alfa) | Epogen® (Amgen®, USA) 150 units/kg intravenous thrice weekly in children < 10 kg | Yes | Symptomatic anaemia in chronic renal failure | Benzyl alcohol | Arthralgia; embolism and thrombosis; headache; hypertension (dose-dependent); influenza-like illness; skin reactions; stroke | Benzyl alcohol administered intravenously in the range of 100 to 200 mg/kg/day has been linked to the “gasping syndrome” [116] |
| Melatonin |
Slenyto® (Neurim Pharmaceuticals®, Israel) 10 mg/day oral in children > 2 years No intravenous preparation—poor water solubility |
Not in UK and Europe Sold over the counter in the USA |
Insomnia with autism spectrum disorder or Smith-Magenis syndrome | In animal experiments: ethanol (Sigma-Aldrich®, USA) and ethanol-free (Chiesi Farmaceutici®, Italy) | Arthralgia; abnormal behaviour; drowsiness; feeling abnormal; headaches; increased risk of infection; altered mood; pain; sleep disorders | Ethanol-free formulation required |
| Cannabidiol |
Epidyolex® (GW Pharmaceuticals®, UK) 20 mg/kg/day oral in children > 2 years No intravenous preparation—poor water solubility |
No | Seizures associated with Lennox-Gastaut syndrome or Dravet syndrome |
In animal experiments: Ethanol (GW Research Ltd.) |
Agitation; appetite abnormal; abnormal behaviour; cough; diarrhoea; drooling; drowsiness; fatigue; fever; increased risk of infection; insomnia; irritability; rash; tremor; vomiting; weight decreased | Ethanol-free formulation undergoing clinical trial |
| Exenatide/exendin-4 | Byetta® (AstraZeneca®, UK) 10 μg twice daily subcutaneous in adults | Yes | Type 2 diabetes mellitus for glycaemic control | Metacresol | Appetite decreased; asthenia; constipation; diarrhoea; dizziness; gastrointestinal discomfort; gastrointestinal disorders; headache; nausea; skin reactions; vomiting |
Metacresol at high doses may be cytotoxic and pro-inflammatory [117] Metacresol-free formulation is required |
| Allopurinol sodium | Aloprim® (Mylan Institutional®, USA) 200 mg/m2/day intravenous. Lyophilised powder reconstituted in saline | Yes | Prophylaxis for hyperuricaemia induced by chemotherapy | Sodium hydroxide | Rash | Irritation to the skin, eyes, mucous membranes (high concentrations), toxic pneumonitis; dermatotoxin; and dysphagia |
*Source: US Food and Drug Administration (FDA) and European Medicines Agency (EMA)