Orphan Medications: An Innovative and Promising Approach against Leishmaniasis.

Over the decades, management of protozoan disease – ‘leishmaniasis’ has been considerably challenging on account of inadequacy in drug efficacy, restricted drug availability, unattainability of treatment by impoverished, and the disease’s endemicity in several countries. At present, ≈ 90 countries are affected by leishmaniasis where visceral leishmaniasis (VL) cases reported are ≈ 30,000 and 1 million cases of cutaneous leishmaniasis (CL) (World Health Organization, 2023). Currently no licensed vaccines are commercially available and therefore, advancement in the area of ‘Orphan Drugs’ offers a promising avenue against leishmaniasis. This article lays down a contemporary foundation on ‘Orphan Drugs’ for treating leishmaniasis – one of the neglected tropical diseases (NTDs). Orphan medications have been considered subsidiary by the Pharma industry. Scientific novelty of this article is emphasis on evaluating ‘Orphan Drugs’ against leishmaniasis. Currently, some notable success stories paving breakthrough moments include, usage of L-AmB (Liposomal amphotericin) at a dose of 18–21 mg/kg for VL and HIV-VL coinfection and 2.5 mg/kg of miltefosine/day for 28 days, and drugs namely fluconazole, ivermectin, mebendazole, suramin, nitazoxanide, melarsoprol, auranofin, etc. in the current scenario. Combinatorial therapies are unquestionably proving supportive as customized approaches. At present, Leishmania fractionated vaccine, recombinant vaccine, DNA vaccine, antigen-cocktail vaccine and chimeric vaccine are in the research and development pipeline proving to be a hope in future. This review delves into a remarkable perspective of orphan medications as pharmaco-therapeutics against NTDs and leishmaniasis in particular. All the treatment breakthroughs and innovations will certainly transform our strategies to defeat leishmaniasis in future.