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Journal of Epilepsy and Clinical Neurophysiology
Print version ISSN 1676-2649
THOME-SOUZA, Sigride and VALENTE, Kette D. R.. Orphan drug: arisement of new concept. J. epilepsy clin. neurophysiol. [online]. 2011, vol.17, n.4, pp.144-147. ISSN 1676-2649. http://dx.doi.org/10.1590/S1676-26492011000400006.
INTRODUCTION: Four new antiepileptic drugs have been approved since 2007, eslicarbazepine acetate, lacosamide, rufinamide and stiripentol. Out of these, two drugs are orphan drugs, that is, drugs specifically designed for the treatment of a specific epileptic syndrome, such as stiripentol for Dravet Syndrome and rufinamide for Lennox-Gastaut Syndrome. OBJECTIVE: to review the efficacy, tolerability and adverse effects of the newly released drugs, especially of both orphan drugs. METHODOLOGY: Studies were selected from electronic data base. Analyses of these studies ascertained the most common, rare and long-term adverse effects, efficacy, and effectiveness, as well as comparison with existing drugs. CONCLUSIONS: The development of specific drugs in the treatment of epileptic syndromes constitutes the cornerstone in the treatment of epilepsy, minimizing the time needed to achieve seizure control, with consequent reduced exposure to the deleterious effects of epilepsy.
Keywords : orphan drug; stiripentol; rufinamide; Dravet Syndrome; Lennox-Gastaut Syndrome.