Ibrutinib Receives Two Oncology Breakthrough Therapy Designations from U.S.
Food and Drug Administration
Investigational oncology agent for B-cell malignancies jointly being developed
by Janssen and Pharmacyclics
RARITAN, N.J., Feb. 12, 2013
RARITAN, N.J., Feb. 12, 2013 /PRNewswire/ -- Janssen Research & Development,
LLC (Janssen), today announced the U.S. Food and Drug Administration (FDA) has
granted Breakthrough Therapy Designations for the investigational oral agent
ibrutinib as a monotherapy for two B-cell malignancies: in patients with
relapsed or refractory mantle cell lymphoma (MCL) who have received prior
therapy, and in patients with Waldenstrom's macroglobulinemia (WM).
Enacted as part of the 2012 FDA Safety and Innovation Act (FDASIA),
Breakthrough Therapy Designation is intended to expedite the development and
review time for a potential new medicine "to treat a serious or
life-threatening disease or condition and preliminary clinical evidence
indicates that the drug may demonstrate substantial improvement over existing
therapies on one or more clinically significant endpoints, such as substantial
treatment effects observed early in clinical development."
"We are pleased that the FDA has granted two Breakthrough Therapy Designations
for ibrutinib as the designation represents a major leap forward in
accelerating drug development timelines," said Paul Stoffels, M.D., Chief
Scientific Officer and Worldwide Chairman, Pharmaceuticals, Johnson & Johnson.
"We are committed to realizing the full potential of ibrutinib for patients
with mantle cell lymphoma, Waldenstrom's macroglobulinemia, as well as other
B-cell malignancies, and will work with Pharmacyclics and the FDA to ensure
the clinical development program for ibrutinib continues to move forward as
quickly as possible."
Janssen Biotech, Inc. and Pharmacyclics entered a collaboration and license
agreement in December 2011 to co-develop and co-commercialize ibrutinib. The
filing for ibrutinib in MCL is expected to be made prior to the end of 2013
and discussions with the FDA about filing in WM continue. The implications of
Breakthrough Therapy Designation cannot be determined at this time. Janssen
and Pharmacyclics are working with the FDA to determine any potential
implications of the Breakthrough Therapy Designations to the ongoing and
planned development activities.
"As an oncology product, ibrutinib receiving the Breakthrough Therapy
Designation is an example of progress and hope for patients fighting a range
of cancers. This designation shows that the FDA is dedicated to using an 'all
hands on deck approach' to work on products that show promise in treating
serious and life-threatening diseases," said Dr. Ellen Sigal, Chair and
Founder of Friends of Cancer Research, a think tank and advocacy organization
based in Washington DC. "The breakthrough pathway that our organization worked
to create is intended to speed up the development and review of treatments
that may demonstrate substantial improvement over existing therapies, and
ibrutinib is a great example of using this new designation to potentially
accelerate patient access to promising treatments."
About Mantle Cell Lymphoma
MCL is an aggressive type of B-cell non-Hodgkin lymphoma (NHL) that usually
occurs in older adults. The disease typically begins in the lymph nodes, but
can spread to other tissues, such as bone marrow and the liver. Ibrutinib
targets the B-cell receptor pathway, an important pathway in malignant B-cell
growth and proliferation. In the United States, there are approximately 5,000
new cases of MCL each year.
About Waldenstrom's Macroglobulinemia
Waldenstrom's macroglobulinemia is rare type of lymphoma. The disease begins
with a malignant change to the B-cell during its maturation so that it
continues to reproduce more malignant B-cells resulting in an overproduction
of monoclonal immunoglobulin M antibody (IgM). WM is a hematologic malignancy
for which no established standard of care - or approved therapeutic - exists.
In the United States, there are approximately 1,500 new cases each year.
Ibrutinib was designed to specifically target and selectively inhibit an
enzyme called Bruton's tyrosine kinase (BTK). BTK is a key mediator of at
least three critical B-cell pro-survival mechanisms occurring in parallel –
regulating apoptosis, adhesion, and cell migration and homing. Through these
multiple actions, BTK helps to direct malignant B-cells to lymphoid tissues,
thus allowing access to a microenvironment necessary for survival.
The effectiveness and safety of ibrutinib alone or in combination with other
treatments is being studied in several B-cell malignancies, including chronic
lymphocytic leukemia/small lymphocytic lymphoma, mantle cell lymphoma, diffuse
large B-cell lymphoma, follicular lymphoma, Waldenstrom's macroglobulinemia
and multiple myeloma. To date, five Phase III trials have been initiated with
ibrutinib and a total of 27 trials are currently registered on
About Janssen Research & Development
At Janssen, we are dedicated to addressing and solving some of the most
important unmet medical needs of our time in oncology, immunology,
neuroscience, infectious diseases and vaccines, and cardiovascular and
metabolic diseases. Driven by our commitment to patients, we develop
innovative products, services and healthcare solutions to help people
throughout the world. Janssen Research & Development and Janssen Biotechare
part of the Janssen Pharmaceutical Companies of Johnson & Johnson. Please
visithttp://www.janssenrnd.comfor more information.
(This press release contains "forward-looking statements" as defined in the
Private Securities Litigation Reform Act of 1995. The reader is cautioned not
to rely on these forward-looking statements. These statements are based on
current expectations of future events. If underlying assumptions prove
inaccurate or unknown risks or uncertainties materialize, actual results could
vary materially from the expectations and projections of Janssen Research &
Development, LLC, any of the other Janssen Pharmaceutical Companies and/or
Johnson & Johnson. Risks and uncertainties include, but are not limited to,
general industry conditions and competition; economic factors, such as
interest rate and currency exchange rate fluctuations; technological advances,
new products and patents attained by competitors; challenges inherent in new
product development, including obtaining regulatory approvals; challenges to
patents; changes in behavior and spending patterns or financial distress of
purchasers of healthcare products and services; changes to governmental laws
and regulations and domestic and foreign health care reforms; trends toward
health care cost containment; and increased scrutiny of the healthcare
industry by government agencies. A further list and description of these
risks, uncertainties and other factors can be found in Exhibit 99 of Johnson &
Johnson's Annual Report on Form 10-K for the fiscal year ended January 1,
2012. Copies of this Form 10-K, as well as subsequent filings, are available
online atwww.sec.gov,www.jnj.comor on request from Johnson & Johnson. None
of the Janssen Pharmaceutical Companies nor Johnson & Johnson undertake to
update any forward-looking statements as a result of new information or future
events or developments).
 PUBLIC LAW 112–144—JULY 9, 2012. U.S. Government Printing Office.
 Waldenstrom Macroglobulinemia. American Cancer Society. Available at:
U.S. Medical Inquiries:
SOURCE Janssen Research & Development, LLC
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