Most new medicines are developed for diseases prevalent in high-income countries, where the potential returns offset the high cost and risk of development.
But this model disadvantages the world’s poorest populations and, according to the World Health Organization, two billion people don’t have access to even the most basic of essential medicines.
Medicines Development for Global Health (MDGH), an Australian biopharmaceutical not-for-profit, is addressing this health inequity — researching, developing and delivering new and improved medicines for diseases that disproportionately affect low- and middle-income countries.
The group is developing two highly impactful medicines — moxidectin and dovramilast — for the treatment of up to eight neglected diseases. And while its current focus is on infectious diseases, MDGH can work on any neglected diseases that don’t traditionally have a commercial market.
MDGH licensed the drug moxidectin from the World Health Organization, who didn’t have the resources to get it registered, recognising it as a really promising molecule that may also be impactful in scabies, which is a big problem in Australia and the Pacific.
Since then, it has registered moxidectin with the United States Food and Drug Administration (US FDA) for the treatment of river blindness, marking the first new FDA-approved treatment for river blindness in the last 20 years.
That earned MDGH a golden ticket, a FDA Priority Review Voucher that incentivises companies to spend on neglected diseases and can be worth hundreds of thousands of dollars.
River blindness: a neglected disease
Caused by the parasitic worm Onchocerca volvulus, river blindness is transmitted from person to person by black flies that breed in fast-flowing rivers, primarily in Africa. The millions of larvae released by the adult parasites invade skin and eyes where they can cause severe manifestations, including permanent blindness, itching and disfiguring skin conditions.
More than 200 million people are at risk of infection and almost all infected people live in 29 African countries.
The WHO and endemic countries aim to eliminate parasite transmission, currently primarily through mass administration of ivermectin.
Merck has donated Ivermectin to endemic countries for more than 30 years, which has significantly reduced the disease burden. However, these existing elimination programs rely on mass drug administration or community-directed treatment, in which the whole eligible population in endemic areas receives ivermectin, regardless of infection status.
It’s clear that new treatment options are needed to accelerate progress toward eliminating parasite transmission.
Moxidectin is a promising treatment option, reducing skin microfilariae levels more profoundly and for longer than ivermectin and is expected to accelerate elimination of parasite transmission.
Ghana approves moxidectin
The Ghana Food and Drugs Authority (FDA) has now approved moxidectin — the first river blindness-endemic country to grant regulatory approval of moxidectin — clearing the way for the planned community pilot treatment implementation program to begin in January 2025 and marking an important step towards elimination.
The trial will take place in the Twifo Atti-Morkwa district, a priority area for moxidectin after higher-than-expected disease prevalence was found following multiple rounds of ivermectin mass administration.
“This approval is the culmination of more than 25 years in the development of moxidectin for the treatment of river blindness and other human diseases,” said MDGH’s Sally Kinrade.
“This is a new model – it is the first time a not-for-profit company has achieved regulatory approval and will deliver a completely novel medicine into an endemic country without the involvement of a multinational pharmaceutical company or generic company partner.”
MDGH will provide moxidectin tablets at cost-plus pricing (without profit) for use in low- and middle-income countries. The group is in discussions with the community of stakeholders that are working to eliminate river blindness to determine how to best use moxidectin to help achieve elimination goals and to inform the potential demand.