La Jolla Pharmaceutical Company Announces Launch of Expanded Access Program for LJPC-501
August 08 2017 - 9:00AM
Business Wire
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.
About LJPC-501
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.
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version on businesswire.com: http://www.businesswire.com/news/home/20170808005554/en/
Company ContactsLa Jolla Pharmaceutical CompanySandra
Vedrick, 858-256-7910Associate Director, Investor Relations &
Human Resourcessvedrick@ljpc.comandDennis M. Mulroy,
858-433-6839Chief Financial Officerdmulroy@ljpc.comorMedia
ContactLifeSci Public RelationsMatt Middleman, M.D.,
646-627-8384matt.middleman@lifescipublicrelations.com
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