First new treatment for river blindness approved by U.S. FDA in 20 years
The United States Food and Drug Administration (FDA) has approved moxidectin as the first new treatment for river blindness in 20 years, adding new ammunition to the fight to eliminate this disabling disease. The FDA decision followed priority review of a new drug application for a neglected tropical disease submitted by the not-for-profit biopharmaceutical company Medicines Development for Global Health (MDGH).
River blindness (onchocerciasis) is caused by the parasitic worm Onchocerca volvulus and transmitted to humans through exposure to repeated bites of infected black flies. Around 200 million people are at risk for the disease that causes severe itching, disfiguring skin conditions and visual impairment, including blindness. More than 99% of infected people live in 31 African countries.
Ivermectin, the only drug currently in use, distributed annually to entire communities living in endemic areas, has significantly reduced river blindness, but the global campaign to eliminate the disease requires also more effective drugs. Recently published findings of a large phase 3 clinical study managed by TDR and conducted in the Democratic Republic of Congo, Ghana and Liberia showed that moxidectin is superior to ivermectin in suppressing the presence of parasites (microfilariae) in the skin that cause the disease and are taken up by blackflies.
“We are delighted about the FDA’s decision,” says TDR Director John Reeder. “It is a milestone toward our vision to have moxidectin made available to African countries to quicken the river blindness endgame.”
Model use of tropical disease priority review voucher (PRV) by not-for-profit
This approval is a step forward for disease elimination strategies as well as innovative financing for more treatments of neglected tropical diseases.
Priority review by the FDA speeds up the new drug application process to six months (compared to the standard 10 months) and approval gives the applicant a transferable voucher (the PRV) that can be used or sold for priority review of another drug.
“This voucher to MDGH fully meets the original spirit of the PRV programme to promote research and development of affordable and accessible drugs for neglected tropical diseases. Without this programme, MDGH would not have been able to raise the funds to complete all of work for the new drug application,” says Dr Reeder.
TDR’s long-term commitment
TDR was instrumental in the evaluation of moxidectin for river blindness over nearly two decades of research and collaboration with academic groups, industry, investigators and river blindness control specialists, WHO country offices and other partners in disease-endemic countries.
“We acknowledge the extraordinary persistence and dedication of the team at TDR, without whom this drug approval would not have happened,” says Mark Sullivan, Founder and Managing Director of MDGH.
TDR began its commitment to river blindness control around 40 years ago, acting as the research arm of the Onchocerciasis Control Programme in West Africa (OCP), and then the African Programme for Onchocerciasis Control (APOC), also hosted by WHO.
This was part of TDR’s long-term commitment to find innovative solutions to developing new drugs for neglected conditions – including the repurposing of drugs from veterinary medicine, the pioneering of public-private product development partnerships, and researching effective strategies to implement public health programmes.
Following withdrawal of its industry partner in 2011, TDR managed completion of the phase 3 clinical trial alone, and searched for a new sponsor to register moxidectin for use in onchocerciasis and ensure its manufacturing, availability and affordability. In 2014 all data at TDR's disposal were licensed to MDGH, which raised funds from the Global Health Investment Fund (GHIF) to complete the work needed to meet the requirements for a new drug application to the FDA.
Next steps toward drug availability in endemic countries
MDGH has been preparing for the next steps toward drug availability in countries for months: “The FDA registration is a real achievement but we still have a number of steps to go until African countries can use moxidectin to accelerate elimination of this disabling disease – and we are counting on TDR and the river blindness research and control communities to work with us, says Mr Sullivan.”
“Our work to bring this medicine to those who need it most continues in earnest,” concluded Mr Sullivan. “The funds from the PRV sale will kick start the next set of activities to provide the community with additional data, including data in younger children.”
“We are here for the full journey – we have committed our skills and resources to play our part in ridding the world of this disease that causes so much suffering and disability,” says Mr Sullivan.