Miltefosine, an FDA-approved drug for the ‘orphan disease’, leishmaniasis

Abstract

Introduction: Leishmaniasis, and most tropical diseases, are rare/orphan diseases in the developed world but not rare worldwide. Classic treatment is with parenteral agents, namely, pentavalent antimony and amphotericin B deoxycholate. Miltefosine is the first oral agent for all forms of leishmaniasis. The target regimen is 2.5 mg/kg/day for 28 days, with a maximum dose of 150 mg/day (one 50 mg capsule each with breakfast, lunch and dinner).

Areas covered: Clinical studies visceral, cutaneous and mucosal leishmaniasis treated with miltefosine.

Expert opinion: The 2014 FDA approval of the miltefosine New Drug Application for visceral, cutaneous and mucosal leishmaniasis was a breakthrough in several ways. In a clinical sense, miltefosine was recognized in the US, after previously being approved in Europe and in the developing world, as the first oral treatment for any form of leishmaniasis. In a regulatory sense, miltefosine was the first drug solely developed for leishmaniasis. In a financial sense, the $125 million sale of miltefosine’s Tropical Disease Priority Review Voucher created a financial incentive for all tropical disease products. Clinical issues remaining post-approval include generalizability of efficacy data generated against the specific species evaluated in registration documents to the large number of species worldwide and tolerance issues partially addressed pre-registration.

Keywords:

• leishmaniasis
• miltefosine
• New Drug Application
• orphan drug
• Tropical Disease Priority Review Voucher

Declaration of interest

J Berman is a consultant to Knight Therapeutics, Inc., the owner of miltefosine. The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.