title: Development status of miltefosine as first oral drug in visceral and cutaneous leishmaniasis.
abstract: An oral treatment for visceral and cutaneous leishmaniasis has been searched for over the last decades. An oral drug would facilitate treatment and lower costs. Oral miltefosine (Zentaris/ASTA Medica AG, Germany), an alkylphosphocholine, is under clinical development for treatment of leishmaniasis. Phase I, II and III clinical trials have been performed in visceral leishmaniasis in India; the overall response rate with 100 mg/day over 4 weeks is 96%. A first clinical trial in New World cutaneous leishmaniasis has shown a final cure rate of 94% at a dose of 150 mg/day over 3 or 4 weeks. Side effects are mainly gastrointestinal (vomiting, diarrhoea). Furthermore, transient elevation of transaminases or urea/creatinine has been observed. The clinical results suggest that miltefosine is the first oral therapy that is effective and safe in visceral and cutaneous leishmaniasis.
authors.affiliation: Zentaris/ASTA Medica AG, Frankfurt am Main, Germany.
authors.forename: C
authors.initials: C
authors.lastname: Fischer
authors.forename: A
authors.initials: A
authors.lastname: Voss
authors.forename: J
authors.initials: J
authors.lastname: Engel
mesh_terms: D000284:Administration, Oral; D000328:Adult; D000981:Antiprotozoal Agents / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic; D004334:Drug Administration Schedule; D004345:Drug Industry; D006801:Humans; D016773:Leishmaniasis, Cutaneous / Q000188:drug therapy*; D007898:Leishmaniasis, Visceral / Q000188:drug therapy*; D010767:Phosphorylcholine / Q000031:analogs & derivatives* / Q000627:therapeutic use*; D014944:World Health Organization
chemical_list: D000981:Antiprotozoal Agents; D010767:Phosphorylcholine; C039128:miltefosine