The European Medicines Agency has approved the first ever drug under its Paediatric Use Marketing Authorisation (PUMA) process, which provides 10 years of data protection for innovative uses of off-patent medicines.
The green light was granted to ViroPharma's Buccolam (midazolam oromucosal solution) as a treatment for prolonged, acute, convulsive seizures in infants, toddlers, children and adolescents, from 3 months to less than 18 years of age.
"Prolonged acute convulsive seizures remain a significant and all-too-common health threat in the paediatric and adolescent populations," commented Prof. Lieven Lagae of University Hospitals KU Leuven in Belgium.
Traditionally these patients have been treated using diazepam suppositories, which though effective "presents certain challenges including social acceptance and difficulties in administration," according to Prof. Lagae.
Buccolam is much easier to administer, and is formulated as a pre-filled solution which is delivered through the lining of the mouth. It is manufactured in age-specific forms so that dosing can be tailored effectively to the patient.
All too often, drugs used in paediatric populations have to be cobbled together from formulations designed for adults, for example by splitting tablets, which can make it difficult to deliver accurate dosing. Added to that, a lack of studies in children often means that doctors have to estimate the most appropriate dose for a child.
The PUMA process was implemented in 2007 to encourage research into paediatric medicines, in the face of widespread off-label use of drugs in children with little supporting clinical evidence.
Earlier this year the EMA said that off-label and unauthorised medicine use still accounted for up to 60% of prescriptions for children in both out-patient and in-patient care, with rates highest among very young children and those with severe conditions.
"The marketing authorisation for Buccolam in Europe is an important step towards addressing the need of parents and carers in the community for a rapidly-acting, effective and socially acceptable rescue medication for emergency treatment to terminate prolonged, acute convulsive seizures in children and adolescents,” said Thierry Darcis, ViroPharma's vice president and general manager, Europe.
ViroPharma said the approval sparks a £10 million ($16.3m) payment to the former owners of Auralis Limited, which originally developed Buccolam.