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Australian company leads fight against blinding disease

04/07/2018By Matthew Woodley
An Australian non-profit biopharmaceutical company has received a major boost in its efforts to rid the world of a blinding disease that more than 200 million people are currently at risk of contracting.

Melbourne-based Medicines Development for Global Health (MDGH) announced it had secured US FDA approval for its river blindness treatment moxidectin, paving the way for its implementation in affected countries in the near future. The disease, caused by a parasitic worm spread via fly bites, can result in severe itching, disfiguring skin conditions and visual impairment, including permanent blindness.

MDGH founder and managing director Mr Mark Sullivan said the FDA's decision should make it easier to gain national approvals in the mostly sub-Saharan countries that are affected, while it could also prove to be a financial windfall for the not-for-profit.

“FDA approval is a momentous achievement for any biopharmaceutical company, but it is a particularly rare and exciting event in the neglected diseases setting,” Sullivan said.

“Achieving FDA approval is a critically important milestone for moxidectin, but our work to bring this medicine to those who need it most continues in earnest.”
Mark Sullivan, MDGH.

“As neglected tropical diseases are endemic in low and middle-income countries, there are limited markets for medicines. Therefore, finding investors willing to support development in these diseases is extremely difficult. However, the introduction of the FDA’s neglected diseases PRV [priority review voucher] program has created a market around neglected diseases.”

The PRV program was established to encourage the development of treatments for neglected diseases, as the voucher – a saleable item – permits the holder to accelerate an FDA review of a new drug application from the standard 10 month period to six months. As this time-saving mechanism has significant value to the pharmaceutical industry, it creates an indirect market for neglected disease treatments and in the past has helped companies generate as much as US$350 million (AU$474.07 m).

MDGH is the first not-for-profit company to register a medicine through the tropical disease PRV program, and the academic who authored a 2006 paper on which the scheme is based said it was exactly what he had in mind when he proposed the program.

“The voucher incentive helped Medicines Development for Global Health attract funding to complete testing and registration for a drug that had been on the shelf. I’m delighted that the voucher program is playing a role in treating patients with river blindness, and one day eliminating the disease,” Duke University’s Professor David Ridley said.

Face A Face

According to Sullivan, the World Health Organisation Special Programme for Research and Training in Tropical Diseases (TDR), and the Global Health Investment Fund were also instrumental in securing the approval.

“It takes a broad community to develop a new medicine. FDA approval represents decades of work by thousands of scientists, disease control specialists, expert advisors, community health workers, funders and study participants,” he said.

Sullivan acknowledged the US$13 million (AU$17.64 m) 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.”

The approval of moxidectin was based on data from two randomised, double blind, active controlled clinical studies. Each study met its respective primary endpoints and showed a statistically significant superiority of moxidectin over the current standard of care, ivermectin, in suppressing the presence of the symptom-causing worm larvae in the skin.

The macrocyclic lactone anthelmintic medicine works by selectively binding to the parasite’s glutamate-gated chloride ion channels, which are vital to the function of invertebrate nerve and muscle cells. However, while it has already been approved for use in patients over 12-years-old, Sullivan said there was still a substantial amount that needed to be done before its benefits were felt.

“Achieving FDA approval is a critically important milestone for moxidectin, but our work to bring this medicine to those who need it most continues in earnest,” Sullivan said.

“MDGH plans 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 disabling disease.”

Some of the expected windfall from the sale of the PRV will be made available to manufacture moxidectin, while the rest has been earmarked for further development of other drugs.

Full results from the Phase III study were published in the January issue of Lancet.

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