Development status of miltefosine as first oral drug in visceral and cutaneous leishmaniasis.

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.