For moxidectin, FDA approval for river blindness was just the beginning

For moxidectin, FDA approval for river blindness was just the beginning

Five years ago, the United States Food and Drug Administration (US FDA) approved moxidectin for the treatment of river blindness (onchocerciasis) in people aged 12 and older. It was the first new medicine to be approved for this devastating disease in more than 30 years. As always, we acknowledge the Special Programme for Research and Training in Tropical Diseases (TDR) for their critical contributions to this milestone.

We remain dedicated to seeing this medicine delivered to the over 200 million people who are at risk of onchocerciasis. We have been working tirelessly since the approval to generate the additional data required to see moxidectin take its place in onchocerciasis treatment guidelines and help accelerate eventual elimination of disease transmission.We are currently running two very large clinical studies, partially funded by the European and Developing Countries Clinical Trials Partnership (EDCTP):

1. A repeat dose study (MDGH-MOX-3001, NCT03876262). This 320-participant study is now fully enrolled, with the final participants enrolled earlier this month.
2. A single-dose endemic-community safety study (MDGH-MOX-3002, NCT04311671). This-12,500 participant study is already over 50% enrolled, and with a new study site recently opening in the Côte d’Ivoire (more below).

A key part of the requirements for World Health Organization guideline inclusion is generating the evidence to support use of moxidectin in children under 12 years of age.  We recently completed a paediatric dose-finding study to determine the optimal dose for children aged 4 to 11 years (MDGH-MOX-1006, NCT03962062). Data from this study will be used to support an application to the US FDA in early 2024 to include treatment of children from 4 years of age in the moxidectin prescribing information.

We continue to explore the potential for moxidectin to treat several other important neglected tropical diseases. We’re evaluating moxidectin for the treatment of scabies, lymphatic filariasis, strongyloides and the soil-transmitted helminths.

Although we have taken a moment this month to reflect on the accomplishment of achieving the FDA approval, we know there is much to do, with many other milestones – small and large – still to come. We remain grateful to the study participants, investigators, clinicians, funders and collaborators who have helped us to get this far and who remain committed to this challenge.

-End