Thursday, April 25, 2019
La Jolla Pharmaceutical Company, a leader in the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for the New Investigational Drug Product, LJPC-0118, for the treatment of severe malaria.
The Breakthrough Therapy designation is an FDA program designed to expedite the development and review of drugs that are intended to treat serious or life-threatening conditions. To qualify for this designation, preliminary clinical evidence must demonstrate that the drug may provide substantial improvement over currently available therapy on at least one clinically significant endpoint.
“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, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. “This designation is a key regulatory milestone and we look forward to working closely with the FDA to further develop LJPC-0118.”
La Jolla plans to file a New Drug Application (NDA) with the FDA in the fourth quarter of 2019 for LJPC-0118. The active pharmaceutical ingredient in LJPC-0118 was demonstrated to be superior to quinine in reducing mortality in patients with severe falciparum malaria infection in two randomized, controlled, clinical studies.
About FDA Breakthrough Therapy Designation
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). The benefits of Breakthrough Therapy designation include eligibility for Priority Review and Rolling Review, plus an organizational commitment involving the FDA's senior managers to provide more intensive guidance regarding the development program. Breakthrough Therapy designation does not change the standards for approval.
LJPC-0118 is La Jolla’s investigational product for the treatment of severe malaria. The active pharmaceutical ingredient in LJPC-0118 was demonstrated to be superior to quinine in reducing mortality in patients with severe falciparum malaria infection in two randomized, controlled, clinical studies. La Jolla plans to file a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2019 for 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.
About La Jolla Pharmaceutical Company
La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. GIAPREZA™ (angiotensin II), formerly known as LJPC-501, was approved by the U.S. Food and Drug Administration (FDA) on December 21, 2017 as a vasoconstrictor indicated to increase blood pressure in adults with septic or other distributive shock. LJPC-0118 is La Jolla’s investigational product for the treatment of severe malaria. LJPC‑401 (synthetic human hepcidin), a clinical-stage investigational product, is being developed for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease, myelodysplastic syndrome and polycythemia vera. For more information, please visit www.ljpc.com.
This press release contains forward-looking statements as defined by the Private Securities Litigation Reform Act of 1995. These statements relate to expectations regarding future events or La Jolla’s future results of operations. These statements are only predictions or statements of current expectations and involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from those anticipated by the forward-looking statements. La Jolla cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties and other factors are described in greater detail in La Jolla’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s website at www.sec.gov. These risks include, but are not limited to, risks relating to: the success of development activities for LJPC-0118 and other product candidates; potential indications for which La Jolla’s product candidates may be developed; the timing, costs, conduct and outcome of clinical studies; risks relating to the development of drug candidates; the anticipated treatment of future clinical data by the U.S. Food and Drug Administration (FDA), EMA and other regulatory authorities, including whether such data will be sufficient for approval; the expected duration over which La Jolla’s cash balances will fund its operations; and other risks and uncertainties identified in our filings with the SEC. Forward-looking statements are presented as of the date of this press release, and La Jolla expressly disclaims any intent to update any forward‑looking statements to reflect the outcome of subsequent events.
Senior Director, Investor Relations & Human Resources
La Jolla Pharmaceutical Company
Phone: (858) 207-4264 Ext: 1135
Dennis M. Mulroy
Chief Financial Officer
La Jolla Pharmaceutical Company
Phone: (858) 207-4264 Ext: 1040