Abstract
BackgroundEpilepsy is a serious chronic health condition with a high morbidity impairing the life of patients and afflicted families. Many epileptic conditions, especially those affecting children, are rare disorders generating an urgent medical need for more efficacious therapy options. Therefore, we assessed the output of the US and European orphan drug legislations.MethodsQuantitative analysis of the FDA and EMA databases for orphan drug designations according to STrengthening the Reporting of OBservational studies in Epidemiology (STROBE) criteria.ResultsWithin the US Orphan Drug Act 40 designations were granted delivering nine approvals, i.e. clobazam, diazepam viscous solution for rectal administration, felbamate, fosphenytoin, lamotrigine, repository corticotropin, rufinamide, topiramate, and vigabatrin. Since 2000 the EMA granted six orphan drug designations whereof two compounds were approved, i.e. rufinamide and stiripentol. In the US, two orphan drug designations were withdrawn. Orphan drugs were approved for conditions including Lennox-Gastaut syndrome, infantile spasms, Dravet syndrome, and status epilepticus. Comparing time to approval for rufinamide, which was approved in the US and the EU to treat rare seizure conditions, the process seems faster in the EU (2.2 years) than in the US (4.3 years).ConclusionOrphan drug development in the US and in the EU delivered only few molecular entities to treat rare seizure disorders. The development programs focused on already approved antiepileptic drugs or alternative pharmaceutical formulations. Most orphan drugs approved in the US are not approved in the EU to treat rare seizures although some were introduced after 2000 when the EU adopted the Orphan Drug Regulation.
Highlights
Epilepsy is a serious chronic health condition with a high morbidity impairing the life of patients and affected families through seizures, hospitalizations, emergency department visits, and medication burden [1, 2]
Orphan drugs were approved for conditions including Lennox-Gastaut syndrome, infantile spasms, Dravet syndrome, and status epilepticus
Comparing time to approval for rufinamide, which was approved in the US and the European Union (EU) to treat rare seizure conditions, the process seems faster in the EU (2.2 years) than in the US (4.3 years)
Summary
Epilepsy is a serious chronic health condition with a high morbidity impairing the life of patients and affected families through seizures, hospitalizations, emergency department visits, and medication burden [1, 2]. Epilepsy comprises a large group of syndromes whereof some meet the criteria for a rare disease according to the World Health Organization (WHO), i.e. a condition affecting less than 65–100 in 100,000 inhabitants [3]. Today clinically available anti-epileptic drugs can control seizures in approximately two-third of patients [5,6,7], in rare seizure conditions such as Lennox-Gastaut or Dravet syndrome long term prognosis is guarded and most patients are refractory to medical treatment [8, 9]. Epilepsy is a serious chronic health condition with a high morbidity impairing the life of patients and afflicted families. We assessed the output of the US and European orphan drug legislations.
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