- The Food and Drug Administration has approved Medicines Development for Global Health’s oral antiparasitic drug moxidectin for the treatment of river blindness in adults and adolescents. MDGH is the first nonprofit to be handed a priority review voucher (PRV) under the tropical disease PRV program.
- The approval is based on two pivotal studies, both of which showed that moxidectin was significantly better than standard-of-care at suppressing the parasite worm larvae in the skin.
- River blindness is caused by the parasitic worm Onchocerca volvulus, and symptoms include skin itching and disfigurement, as well as visual impermanent or blindness. Around 120 million people worldwide are at risk of the disease, with 96% in Africa.
Tropical diseases, including parasitic infections, are particularly problematic in low- and middle-income countries, where there is little budget for drugs and healthcare, and as such few incentives for companies and financiers to invest in the field. To boost the market, the Food and Drug Administration created its PRV program, which awards the valuable vouchers to companies that successfully develop treatments for tropical or rare pediatric illnesses.
Each voucher can then be used on a future drug project to cut the regulatory review time from the standard 10 months to six months.
“This voucher to MDGH exemplifies the original spirit of the program — to create incentives for research and development in neglected diseases,” said John Reeder, director of the World Health Organization Special Program for Research and Training in Tropical Diseases (TDR), in a June 13 statement.
While helpful for other pipeline programs, PRVs can also be sold — making them a very valuable currency in the pharma world. In April 2018, Spark Therapeutics sold a PRV to Jazz Pharmaceuticals for $110 million. That is well below the record-breaking PRV transaction between AbbVie and United Therapeutics in 2015, which totaled $350 million, yet still showcases how PRVs can be a valuable cash injection.
For a nonprofit like MDGH, focused on developing affordable medicines and vaccines for low-income countries, such an injection would be quite powerful.
The moxidectin research at MDGH was supported by WHO’s TDR, and by a $13 million investment from the Global Health Investment Fund. Moxidectin research is ongoing too: in river blindness in children; in the treatment and prevention of scabies; in the treatment of worm infections; and in lymphatic filariasis.
“FDA approval is a momentous achievement for any biopharmaceutical company, but it is a particularly rare and exciting event in the neglected diseases setting,” Mark Sullivan, founder and managing director of MDGH, said in the June 13 statement.