Over the past two decades, global efforts to address neglected tropical diseases (NTDs) have made great strides. Large-scale treatment programmes and unprecedented public-private partnerships have considerably reduced disease burden, improved people’s health and well-being, and changed the outlook for several NTDs in entire communities.

These public health gains are significant. Yet, they also highlight a growing constraint at the core of the NTD response: without new, safe and efficacious drugs or sustained pipelines to develop them, progress against NTDs will stall.

For many NTDs, available treatment strategies still rely on a single or only a small number of drugs.¹ These drugs have been essential in reducing disease burden, but their limited effectiveness, repeated use and lack of alternatives expose a vulnerability in the current approach. Drug discovery and development are therefore no longer peripheral concerns; they are central to sustaining progress.

Where current drugs fall short

Anthelminthic drugs illustrate this challenge clearly. Parasitic worm infections affect an estimated 1.5 billion people worldwide, particularly those living in poverty. These infections are caused by different species of worms, including whipworms, hookworms, and roundworms, the three main soil-transmitted helminths responsible for substantial morbidity.

Current treatment options are limited. Albendazole and mebendazole continue to form the backbone of therapy for soil-transmitted helminth infections, despite well-documented shortcomings, including low efficacy against whipworm infections.²

Dependence on such a small set of drugs also heightens concern about resistance, particularly given their widespread and repeated use. Together, these factors reveal how fragile progress can become when therapeutic options remain narrow.

Building a pipeline, not a single product

When it comes to drug discovery and development for NTDs, markets are deficient, development timelines are long, and failure rates are high. As a result, parasitic worm infections – and many other NTDs – have historically attracted limited investment from the pharmaceutical industry and research and development (R&D) more generally.³

One way forward is to expand treatment options through repurposing and combination therapy.⁴ Emodepside is one such example.⁵ Originally developed for veterinary use, it is now being evaluated for human parasitic worm infections. Clinical studies have demonstrated high efficacy and a good safety profile.^6,7 Two pivotal phase III trials were initiated in late 2025 in collaboration with Bayer, the Public Health Laboratory Ivo de Carneri, and the University of the Philippines.

If development continues as expected, emodepside could become the first new broad-spectrum anthelminthic drug approved for human use in more than four decades; a rare milestone and a reminder that sustained, long-term investment and product development partnerships can deliver meaningful advances.

Progress also depends on platforms designed to sustain multiple candidates rather than single breakthroughs. The EDCTP-funded Helminth Elimination Platform (HELP) is one such approach.⁸ Led by Swiss TPH, HELP brings together academic institutions, non-profit organisations, and pharmaceutical companies to advance novel drugs for parasitic worm infections, including hookworm, whipworm, onchocerciasis and lymphatic filariasis.

HELP spans drug discovery through early clinical development, including the first-in-human phase I trials in Africa, while contributing to research capacity strengthening in countries affected by these diseases. Results from the phase I studies enabled progression into phase II clinical research and supported the successful acquisition of additional funding through the EDCTP-funded eWHORM programme.