The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use(CHMP) has recommended granting two new paediatric-use marketing authorisations (PUMAs), for Kigabeq (vigabatrin) and Slenyto (melatonin).
PUMAs can be granted for authorised medicines which have been developed specifically for children and are no longer under patent protection. PUMAs aim to stimulate research of existing medicines for better treatments for children by giving the medicines ten years of market protection.
Slenyto is to be used for the treatment of insomnia in children from 2 years of age with autism spectrum disorder and Smith-Magenis syndrome (a disorder with a variety of features including intellectual disability, speech and language delay, distinctive facial features, difficulty sleeping and behavioural problems). Sleep disorders are common in children with developmental disabilities and are often difficult to treat. There are currently no approved medicines to treat insomnia in children. However, in practice doctors have been prescribing medicines off-label, including melatonin.
Data from the clinical trial and from the scientific literature suggest the medicine is associated with a significant increase in total sleep time, a shortened sleep latency (the time it takes to fall asleep after the lights have been turned out) and a longer duration of uninterrupted sleep. The main side effects observed in the clinical trial were somnolence (sleepiness), headache and fatigue.
Kigabeq, the other medicine recommended by the CHMP at this meeting, is meant for the treatment of infantile spasms (West's syndrome), an uncommon and severe form of epilepsy associated with a highly-resistant seizure type (epileptic spasms) and a rapid psychomotor regression, and in resistant partial epilepsy, in infants and children from 1 month to 7 years of age.
Vigabatrin, the active substance in Kigabeq, is an antiepileptic agent that was first authorised in European countries almost thirty years ago and is commonly used to treat adult and paediatric patients, in particular patients with forms of epilepsy which are difficult to manage. Currently vigabatrin is available in the European Union (EU) as 500 mg film-coated tablets or granules for oral solution sachets, which need to be split and/or diluted to make them appropriate for children.
Kigabeq has been developed as 100 mg and 500 mg soluble tablets, with incremental unitary doses of 50 mg, to allow better adjustment of the dose to the patient’s body weight.
