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La Jolla Pharmaceutical Company (NASDAQ: LJPC) (the Company or La Jolla), today announced that the Company has initiated an expanded access program (EAP) in the United States to provide its investigational drug, LJPC-501 (angiotensin II), to patients with vasodilatory or distributive shock who cannot achieve target mean arterial pressure (MAP) despite adequate fluid resuscitation and treatment with currently available vasopressors (a syndrome alternatively known as “catecholamine resistant hypotension” or “clinically refractory hypotension” (CRH)).
Expanded access, sometimes known as compassionate use, is an option facilitated by the U.S. Food and Drug Administration (FDA) to make available prior to regulatory approval investigational medicine(s) for the treatment of serious or life-threatening diseases or conditions where there are no ongoing clinical trials and there is a lack of satisfactory therapeutic alternatives.
“We are pleased to commence this expanded access program for eligible patients in the U.S.,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “This EAP provides a mechanism for eligible patients to access LJPC-501 while the Company is diligently working to pursue FDA approval."
About LJPC-501 Expanded Access Program
The LJPC-501 EAP is a program for U.S. patients 18 years of age or older with vasodilatory or distributive shock who are unable to achieve target MAP despite adequate fluid resuscitation and treatment with currently available vasopressors. The enrollment criteria are available at www.clinicaltrials.gov.
Expanded access, sometimes called "compassionate use," is the use of an investigational medical product to treat a patient’s serious or life-threatening disease or condition outside of a clinical trial. The main distinction between expanded access and the use of an investigational medicine in clinical studies is that expanded access programs are not intended to obtain information about the safety or effectiveness of the drug. Expanded access to an investigational medicinal product can only be provided if the sponsor is actively pursuing marketing approval of the drug. The investigational medicine(s) made available through expanded access have not yet received regulatory approval; therefore, their potential safety and efficacy have not yet been established by the FDA. Doctors and patients should consider all possible benefits and risks when seeking access to investigational medicine(s) prior to regulatory approval.
Under 21 CFR 312.305(a)(3), to authorize any category of expanded access, FDA must determine that expanded access to the drug for the requested use will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of the expanded access use or otherwise compromise the potential development of the drug for the expanded access use. In addition, FDA must determine that there is sufficient evidence of the safety and effectiveness of the investigational product to support its use in the particular circumstance.
FDA guidance on expanded access can be found here.
Once a regulatory agency approves the investigational medicine for commercial use and the medicine is commercially available, existing expanded access programs for that medicine will be phased out as soon as practicable while not interfering with patient safety.
LJPC-501 is La Jolla’s proprietary formulation of synthetic human angiotensin II. Angiotensin II, the major bioactive component of the renin-angiotensin system, serves as one of the body’s central regulators of blood pressure. LJPC-501 is being developed for the treatment of patients with catecholamine resistant hypotension (CRH). LJPC-501 is the first synthetic human angiotensin II product candidate to be tested in a Phase 3 study. In February 2017, La Jolla reported positive top-line results from the ATHOS-3 (Angiotensin II for the Treatment of High-Output Shock) Phase 3 study of LJPC-501 in patients with CRH. In May 2017, the results of ATHOS-3 were published by The New England Journal of Medicine in an article entitled “Angiotensin II for the Treatment of Vasodilatory Shock.” The NEJM article and its Supplementary Appendix can be found here.
About Catecholamine Resistant Hypotension
Catecholamine resistant hypotension or clinically refractory hypotension (CRH) is a life-threatening syndrome in patients with vasodilatory (also known as distributive) shock (dangerously low blood pressure with adequate cardiac function) who cannot achieve target mean arterial pressure (MAP) despite adequate fluid resuscitation and treatment with currently available vasopressors (catecholamines and/or vasopressin). There are approximately 500,000 distributive shock cases in the United States per year, an estimated 200,000 of which develop CRH. On current therapies, more than 50% of CRH patients die within 30 days.
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. The Company has several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of synthetic human angiotensin II for the potential treatment of catecholamine resistant hypotension. LJPC-401 is La Jolla’s proprietary formulation of synthetic human hepcidin for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. LJPC-30S is La Jolla’s next-generation gentamicin derivative program that is focused on the potential treatment of serious bacterial infections as well as rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. For more information on La Jolla, please visit www.ljpc.com.
Forward Looking Statement Safe Harbor
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or the Company’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. The Company 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 the Company’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site www.sec.gov. These risks include, but are not limited to, risks relating to: the timing and availability of LJPC-501 under the expanded access program; the timing and prospects for approval of LJPC-501 by the FDA and the other regulatory authorities; risks relating to the scope of product labels (if approved) and potential market sizes, as well as the broader commercial opportunity; the anticipated timing for regulatory actions; and the success of future development activities; potential indications for which the Company’s product candidates may be developed. The Company expressly disclaims any intent to update any forward-looking statements to reflect the outcome of subsequent events.
La Jolla Pharmaceutical Company
Sandra Vedrick, 858-256-7910
Associate Director, Investor Relations & Human Resources
Dennis M. Mulroy, 858-433-6839
Chief Financial Officer
LifeSci Public Relations
Matt Middleman, M.D., 646-627-8384
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