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

An oral treatment for visceral and cutaneous leishmaniasis has been searche d for over the last decades. An oral drug would facilitate treatment and lo wer costs. Oral miltefosine (Zentaris/ASTA Medica AG, Germany), an alkylpho sphocholine, is under clinical development for treatment of leishmaniasis. Phase I, II and III clinical trials have been performed in visceral leishma niasis 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 e ffects are mainly gastrointestinal (vomiting, diarrhoea). Furthermore, tran sient 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.