Orphan medicines: special treatment required?: case study 2: Diacomit and severe myoclonic epilepsy in infancy

Document type
Case study
Corporate author(s)
2020health (Think tank)
Publisher
2020health
Date of publication
1 November 2012
Subject(s)
Health Services
Collection
Social welfare
Material type
Reports

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This case study on the drug Diacomit and treatment of severe myoclonic epilepsy in infancy accompanies a report that examines the evidence to see if there are ways of improving decision making on treatments for people with rare diseases in the NHS. Diacomit (stiripentol) marketed by Laboratoires Biocodex, is an antiepileptic, or anticonvulsant, medicine. It has been shown in clinical trials to be useful as an add-on to clobazam and valproate (and other antiepileptic medicines) for children with a very rare type of epilepsy called severe myoclonic epilepsy in infancy (SMEI), also known as Dravet’s syndrome. Together treatments can help reduce the number of seizures.

Diacomit was awarded orphan drug status for its use in SMEI in 2001, reflecting the small number of patients with SMEI. Marketing approval followed in 2007, with Diacomit being authorised by European Medicines Agency for use in conjunction with clobazam and valproate as adjunctive therapy of refractory generalized tonicclonic seizures in patients with severe myoclonic epilepsy in infancy (SMEI or Dravet's syndrome) whose seizures are not adequately controlled with clobazam and valproate. Diacomet is perceived as fulfilling a treatment gap when previous treatments have been unsuccessful.

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