EMA Innovation Task Force Meeting
Granted
La Jolla Pharmaceutical Company (Nasdaq: LJPC) (the "Company" or
"La Jolla"), a leader in the development of innovative therapies
intended to significantly improve outcomes in patients suffering
from life-threatening diseases, today announced that the European
Medicines Agency (EMA) Committee for Orphan Medicinal Products
(COMP) issued a positive opinion recommending LJPC-401, La Jolla’s
novel formulation of hepcidin, for designation as an orphan
medicinal product for the treatment of chronic iron overload
requiring chelation therapy. Chronic iron overload occurs in
patients suffering from beta thalassemia, sickle cell disease and
hereditary hemochromatosis (HH). The final opinion, which is
subject to review and approval by the European Commission (EC), may
include all or a subset of these conditions.
Beta thalassemia and sickle cell disease are genetic diseases of
blood cells that can cause life-threatening anemia and often
require frequent and life-long blood transfusions. These blood
transfusions, while necessary to treat anemia, cause excessive iron
accumulation in the body, which is toxic to vital organs. HH is a
disease caused by a genetic deficiency in hepcidin production,
resulting in excessive iron accumulation. HH is the most common
genetic disease in Caucasians and causes liver cirrhosis, liver
cancer, heart disease and/or failure, dementia and diabetes.
Iron chelators are drugs that bind to and help clear excessive
iron from the body. However, chelators cause significant toxicity,
including kidney failure, liver failure or gastrointestinal
hemorrhage.
LJPC-401 is La Jolla’s novel formulation of hepcidin, a
naturally occurring peptide hormone that is the body’s regulator of
iron absorption and distribution. Hepcidin prevents excessive iron
accumulation in organs, such as the liver and heart, where it can
cause significant damage and even result in death. La Jolla is
developing LJPC-401 for the treatment of iron overload, which
occurs as a result of diseases such as HH, beta thalassemia and
sickle cell disease.
In a separate decision, the EMA’s Innovation Task Force (ITF)
has granted a meeting with La Jolla to review future development
plans for LJPC-401. The ITF provides an interactive platform for
applicants with novel and innovative therapies to proactively
discuss the scientific, legal and regulatory aspects of development
for such therapies with the EMA. This early dialogue is intended to
contribute to the preparedness of an applicant and to increase EMA
awareness and education of emerging therapies and technologies.
“We believe that LJPC-401 can have a major impact on the lives
of patients suffering from chronic iron overload,” said George F.
Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La
Jolla. “We are encouraged by the positive feedback and support of
the EU regulatory authorities and are excited to further our
clinical development of LJPC-401.”
Emas Pharma Limited served as La Jolla’s European regulatory
representative for these regulatory interactions.
About European Orphan Drug Designation
Orphan drug designation is a status assigned to a medicine
intended for use in rare diseases. To be granted orphan status in
the European Union (EU), the medicine must be for the treatment of
a life-threatening or chronically debilitating condition that
affects no more than five in 10,000 people in the EU and for which
no satisfactory treatments exist or, where they do exist, the
medicine will be of significant benefit to those affected by that
condition.
Applications for orphan designation are evaluated by the
European Medicines Agency’s (EMA) Committee for Orphan Medicinal
Products (COMP), which provides its opinion on whether or not the
medicine qualifies as an orphan medicine for the treatment,
prevention or diagnosis of a rare disease. If the COMP issues a
positive opinion, the European Commission (EC) may then grant the
medicine orphan status.
An orphan designation allows a pharmaceutical company to benefit
from incentives from the EU to develop a medicine for a rare
disease, such as reduced fees, regulatory support during the
product development phase, access to the centralized authorization
procedure (a single application for all EU countries), and 10 years
of market exclusivity once the medicine is approved.
About the European Medicines Agency’s Innovation Task
Force
The European Medicines Agency (EMA) established its Innovation
Task Force (ITF) in order to provide support for medical innovation
in the European Union (EU), with a particular focus on emerging
therapies and technologies. The ITF provides an interactive
platform for applicants with novel and innovative therapies to
proactively discuss the scientific, legal and regulatory aspects of
development for such therapies with the EMA. This early dialogue is
intended to contribute to the preparedness of an applicant and to
increase EMA awareness and education of emerging therapies and
technologies.
About Beta Thalassemia
Beta thalassemia is a disease characterized by a genetic
mutation that results in the underproduction of hemoglobin, the
body’s natural oxygen-carrying molecule contained in red blood
cells. There are three types of beta thalassemia: beta thalassemia
minor, beta thalassemia intermedia and beta thalassemia major.
Patients with the more severe forms (intermedia and major) suffer
from severe and sometimes life-threatening anemia, bone deformities
and enlargement of the spleen. Standard treatment includes
frequent, life-long blood transfusions. While lifesaving, these
transfusions cause an excess of iron accumulation, which in turn is
toxic to vital organs, such as the liver and heart. The only
currently approved treatments for iron overload are iron chelators,
which may cause kidney failure, liver failure or gastrointestinal
hemorrhage.
About Sickle Cell Disease
Sickle cell disease is the most common inherited blood disorder
in the United States. Sickle cell disease is characterized by a
genetic mutation that results in the production of abnormal
hemoglobin, the body’s natural oxygen-carrying molecule contained
in red blood cells. The abnormal hemoglobin causes the red blood
cells to form a “sickle,” or crescent, shape. Patients with severe
forms suffer from severe and sometimes life-threatening anemia,
strokes, and damage to vital organs such as the lungs, spleen,
kidney and liver. Standard treatment includes frequent, life-long
blood transfusions. While lifesaving, these transfusions cause an
excess of iron accumulation, which in turn is toxic to vital
organs, such as the liver and heart. The only currently approved
treatments for iron overload are iron chelators, which may cause
kidney failure, liver failure or gastrointestinal hemorrhage.
About Hereditary Hemochromatosis
Hereditary hemochromatosis (HH) is the most common genetic
disease in Caucasians. HH is a disease characterized by a genetic
mutation that results in a deficiency in the production of
hepcidin, which is the body’s naturally occurring regulator of iron
absorption and distribution. Without proper levels of hepcidin,
excessive amounts of iron accumulate in the body and can lead to
liver cirrhosis, liver cancer, heart disease and/or failure,
dementia and diabetes. With no FDA-approved treatments for HH,
patients are treated with iron chelators and phlebotomy, which do
not address the underlying disease pathology, carry significant
toxicity and/or adversely impact quality of life.
About LJPC-401
LJPC-401 is La Jolla’s novel formulation of hepcidin. Hepcidin,
an endogenous peptide hormone, is the body’s naturally occurring
regulator of iron absorption and distribution. Hepcidin prevents
excessive iron accumulation in tissues, such as the liver and
heart, where it can cause significant damage and even result in
death.
La Jolla is developing LJPC-401 for the treatment of iron
overload, which occurs as a result of diseases such as hereditary
hemochromatosis (HH), beta thalassemia and sickle cell disease. HH
is a disease caused by a genetic deficiency in hepcidin that
results in excessive iron accumulation. HH is the most common
genetic disease in Caucasians and causes liver cirrhosis, liver
cancer, heart disease and/or failure, dementia and diabetes. Beta
thalassemia and sickle cell disease are genetic diseases of the
blood that can cause life-threatening anemia and usually require
frequent and life-long blood transfusions. These blood transfusions
cause excessive iron accumulation in the body, which is toxic to
vital organs, such as the liver and heart.
LJPC-401 has been shown to be effective in reducing serum iron
in preclinical testing. La Jolla’s Investigational New Drug
Application (IND) has been accepted by the FDA, and La Jolla
expects to release preliminary results from a Phase 1 study by the
end of 2015.
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 angiotensin II for the potential
treatment of catecholamine-resistant hypotension. LJPC-401 is La
Jolla’s novel formulation of hepcidin for the potential treatment
of iron overload, which occurs as a result of diseases such as
hereditary hemochromatosis (HH), beta thalassemia and sickle cell
disease. LJPC-30Sa and LJPC-30Sb are La Jolla’s next-generation
gentamicin derivatives for the potential treatment of serious
bacterial infections and 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 document 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 and
involve known and unknown risks, uncertainties and other factors,
which may cause actual results to be materially different from
these 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 for the filing of
an Investigational New Drug Application (IND), commencement of
clinical studies and the anticipated timing for completion of such
studies; the success of future development activities for the
Company’s drug candidates; potential indications for which the
Company’s drug candidates may be developed; and the Company’s
ability to obtain the potential benefits of orphan drug designation
for its drug candidates. Subsequent written and oral
forward-looking statements attributable to the Company or to
persons acting on its behalf are expressly qualified in their
entirety by the cautionary statements set forth in the Company’s
reports filed with the SEC. The Company expressly disclaims any
intent to update any forward-looking statements.
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version on businesswire.com: http://www.businesswire.com/news/home/20150911005176/en/
La Jolla Pharmaceutical CompanyGeorge F. Tidmarsh, M.D.,
Ph.D.President & Chief Executive
Officer858-207-4264gtidmarsh@ljpc.comandDennis M. MulroyChief
Financial Officer858-433-6839dmulroy@ljpc.com
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