La Jolla Pharmaceutical Company has been granted Orphan Drug Designation for the Investigational Drug Product, LJPC-0118, for the treatment of severe cases of malaria. The Active Pharmaceutical Ingredient (API) is artesunate. Artesunate demonstrated superiority over quinine in reducing the mortality rates of patients suffering from severe falciparum malaria infection. This was tested in two controlled, clinical trials. Being granted Orphan Drug Designation by the Food and Drug Administration (FDA) is a significant milestone in the company’s effort to come up with a cure for malaria.The FDA grants orphan status to drugs that are intended for the treatment, prevention or diagnosis of rare diseases or disorders which affect less than 200,000 people in the U.S. If a
sponsor receives regulatory approval from the FDA, they are granted seven years of marketing exclusivity and development incentives including tax credits related to clinical trial expenses and FDA assistance in clinical trial design.
“The granting of orphan drug status is a significant milestone for the LJPC-0118 program and highlights the significant unmet medical need for the treatment of malaria,” said George Tidmarsh, La Jolla’s President and Chief Executive Officer. “There are currently no approved treatments in the U.S. for patients with severe malaria. We are preparing to file a new drug application later this year and, upon approval, to making this front-line treatment for severe malaria available to the public.”
Severe malaria is a sometimes fatal disease caused by a parasite that infects a certain type of mosquito. The most common symptoms include fever, hypoglycemia, chills, sweating, and shock. Usually, the Central Nervous System gets infected which may result in a coma. In 2017, an estimated 219 million cases of malaria occurred worldwide, out of which 435,000 people succumbed to the disease.