La Jolla Pharmaceutical has secured breakthrough therapy designation from the US Food and Drug Administration (FDA) for its investigational compound, LJPC-0118, to treat severe malaria.
Severe malaria is a serious condition characterised by fever, chills, sweating, hypoglycemia and shock. The disease is commonly accompanied by central nervous system infections that may lead to delirium, which may eventually result in coma.
When tested in two randomised, controlled clinical studies, the active pharmaceutical ingredient in LJPC-0118 showed superiority over quinine in mitigating mortality in patients with severe falciparum malaria infection.
The breakthrough therapy designation is set to speed up the development and review of the potential drug. It also provides eligibility for priority review and rolling review by the regulator.
La Jolla Pharmaceutical noted that breakthrough status is granted when preliminary clinical evidence suggests that the product may exhibit substantial improvement over current treatments on a minimum of one clinically significant endpoint.
The company is planning to submit a new drug application (NDA) to the FDA for LJPC-0118 in severe malaria in the fourth quarter of this year.
La Jolla Pharmaceutical president and CEO George Tidmarsh said: “We are committed to the rapid development of LJPC-0118 to help patients suffering from severe malaria, a serious and life-threatening disease without a current FDA-approved therapy.
“This designation is a key regulatory milestone and we look forward to working closely with the FDA to further develop LJPC-0118.”
Statistics show that more than 219 million people were diagnosed with malaria in 2017 globally, including an estimated 200 million cases in the World Health Organization (WHO) African region.
During the year, malaria was responsible for approximately 435,000 deaths worldwide.