La Jolla Pharmaceutical’s LJPC-0118 (artesunate) has been granted orphan drug designation from the US Food and Drug Administration (FDA) for the treatment of malaria.
According to the California-based biopharma company, artesunate – the active pharmaceutical ingredient in LJPC-0118 n two randomized, controlled, clinical studies was shown to be superior to quinine in lowering mortality in patients suffering from severe falciparum malaria infection.
Under the FDA orphan drug designation program, drugs and biologics, which are defined as those developed for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that impact lesser than 200,000 people in the US, get the status.
The orphan drug status gives up to seven years of marketing exclusivity if the sponsor becomes the first to get regulatory approval from the FDA. The designation also provides multiple development incentives such as tax credits associated with clinical trial expenses, FDA assistance in clinical trial design, and an exemption from the FDA-user fee.
George Tidmarsh – La Jolla President and CEO said: “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.
“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.”
La Jolla is looking to file a new drug application (NDA) with the FDA for LJPC-0118 in the fourth quarter of 2019.