Which of the following is granted orphan drug status for the treatment of Dravet's syndrome?: (A) Stiripentol, (B) Icatibant, (C) Pitolisant, (D) Tafamidis
Answer is A. Stiripentol increases GABA transmission. It is granted orphan drug status for the treatment of Dravet's syndrome (Severe myoclonic epilepsy in infants).Icatibant is a bradykinin B2 receptor antagonist. It is used for acute attacks of hereditary angioedema in adults with C1-esterase inhibitor deficiency.Pitolisant (a/k/a Tiprolisant) is an inverse agonist of an H3 receptor. It is under trial for schizophrenia and Parkinson's disease.Tafamidis stabilizes the correctly folded tetrameric form of TTR. It is used in familial amyloid polyneuropathy.