FDA grants breakthrough therapy status to treatment for severe malaria

The new investigational drug product developed by La Jolla Pharmaceutical Company has received the US FDA Breakthrough Therapy designation for the treatment of severe malaria.

The active pharmaceutical ingredient in the treatment LJPC-0118 demonstrated superiority to quinine in reducing mortality in patients with severe falciparum malaria infection in two randomized trials. Based on these results, the pharmaceutical company plans to submit a New Drug Application with the FDA in the fourth quarter of 2019.

“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. We are very pleased to receive the Breakthrough Therapy designation from the FDA and are committed to providing LJPC-0118 to patients in the US,” said George Tidmarsh, La Jolla’s President, and Chief Executive Officer, said in a press release. "This designation is a key regulatory milestone and we look forward to working closely with the FDA to further develop LJPC-0118.”

Severe malaria is a serious and sometimes fatal disease caused by a parasite that commonly infects a certain type of mosquito, which feeds on humans. Symptoms include but are not limited to: fever, chills, sweating, hypoglycemia, and shock. Severe malaria is often complicated by central nervous system infections that may lead to delirium, which may progress to coma. Infections usually occur a few weeks after being bitten.

In 2017, an estimated 219 million cases of malaria occurred worldwide, with an estimated 200 million of these cases occurring in the World Health Organization (WHO) African Region, and, in 2013, the global annual incidence of severe malaria was estimated to be 2 million cases. In 2017, an estimated 435,000 people died from malaria worldwide.

The FDA's Breakthrough Therapy designation is a process designated to expedite the development and review of a drug candidate that is planned for use, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoint(s).