Medicines Development for Global Health (MDGH) and the World Health Organization Special Program for Research and Training in Tropical Diseases (TDR) announced that the US Food and Drug Administration (FDA) has approved moxidectin 8 mg oral for the treatment of onchocerciasis (river blindness) in patients aged 12 years and older.
"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 MDGH, in a recent statement
“It takes a broad community to develop a new medicine,” he added. “FDA approval represents decades of work by thousands of scientists, disease control specialists, expert advisors, community health workers, funders and study participants. We particularly acknowledge the US$13 million investment from the Global Health Investment Fund (GHIF) as well as the extraordinary persistence and dedication of the team at TDR, without whom this would not have happened."
Onchocerciasis occurs from a parasitic worm, onchocerca volvulus
, and is characterized by severe itching, disfiguring skin conditions, and visual impairment that includes permanent blindness as a result of the worm's larvae (microfilariae).
Data from 2 randomized, double blind, active controlled clinical studies served as the basis for the approval. Respective primary endpoints were met in each study, which displayed a statistically significant superiority of moxidectin over ivermectin, which is the current standard of care, in suppressing the microfilariae’s presence in the skin. The phase III study’s full results were published in the Lancet
in January 2018.
"We are delighted about the FDA's decision," added the UNICEF/UNDP/World Bank/WHO Special Program for Research and Training in Tropical Diseases (TDR) Director, John Reeder. "It is a milestone toward the river blindness endgame and our objective to enable African countries to integrate moxidectin into their elimination strategies."
In addition, the FDA awarded MDGH a priority review voucher (PRV). MDGH is the first not‑for‑profit company to register a medicine through the tropical disease PRV program.
Reeder further explained how moxidectin’s approval was enabled by the PRV program is the result of a paradigm-changing approach to the development of new medicines for neglected diseases. He added that since limited medicinal markets exist for neglected and endemic tropical diseases as a result of their populating in low and middle-income countries, locating investors to support drug development in these diseases is challenging.
However, the FDA’s neglected disease PRV program has opened a market surrounding neglected diseases. In order to encourage the development of new drug and biological products for neglected diseases, the PRV legislation was designed. The PRV allows the holder to prioritize the review of a new drug application (NDA) from the standard 10 months to 6 months.