After a priority review, the FDA has cleared the use of moxidectin, the first new treatment for river blindness (onchocerciasis) in 20 years. The oral drug is indicated for patients aged 12 years or older.
The decision comes on the heels of 2 clinical trials demonstrating the superiority of moxidectin over the current standard treatment, ivermectin.
"FDA approval is a momentous achievement for any biopharmaceutical company, but it is a particularly rare and exciting event in the neglected diseases setting," stated Mark Sullivan, founder and managing director of the not-for-profit pharmaceutical company Medicines Development for Global (MDGH).
Co-developed by MDGH and World Health Organization Special Programme for Research and Training in Tropical Diseases (TDR), moxidectin’s approval marks a step forward in eliminating this rare disease that almost exclusively afflicts sub-Saharan Africa. The region is a breeding ground for the black flies that carry and transmit the parasitic worm responsible for river blindness, Onchocerca volvulus. Once in the human host, the parasite can damage all ocular tissue, leading to corneal sclerosing keratitis, secondary glaucoma, cataracts, secondary glaucoma, chorioretinitis and optic atrophy.
Though ivermectin is effective, TDR researchers have been working since the late 1990s to develop a superior drug that could help eradicate the disease. In both randomized, double-masked, controlled clinical trials, one of which was published in the January 2018 issue of The Lancet, moxidectin (8 mg) was significantly better at reducing the parasite load found in patients’ skin compared with ivermectin (150 μg/kg).
With the regulatory clearance under its belt, MDGH is now working to make moxidectin accessible to everyone.
"MDGH plans to provide the community with additional data, including data in younger children,” elaborated Sullivan. “We are here for the full journey – we have committed our skills and resources to play our part in ridding the world of this disabling disease."
Image courtesy of Wikimedia Commons: F. Christian Thompson.