ARIAD Announces That Iclusig™ (Ponatinib) Receives Positive CHMP Opinion for
Approval in the European Union
CAMBRIDGE, Mass. & LAUSANNE, Switzerland -- March 22, 2013
ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) today announced that the Committee
for Human Medicinal Products (CHMP) of the European Medicines Agency (EMA) has
adopted a positive opinion on the marketing authorization application for
Iclusig™ (ponatinib) for two indications:
*The treatment of adult patients with chronic phase, accelerated phase or
blast phase chronic myeloid leukaemia (CML) who are resistant to dasatinib
or nilotinib; who are intolerant to dasatinib or nilotinib and for whom
subsequent treatment with imatinib is not clinically appropriate; or who
have the T315I mutation, or
*The treatment of adult patients with Philadelphia-chromosome positive
acute lymphoblastic leukaemia (Ph+ ALL) who are resistant to dasatinib;
who are intolerant to dasatinib and for whom subsequent treatment with
imatinib is not clinically appropriate; or who have the T315I mutation.
“The positive recommendation by the CHMP of Iclusig for use in CML and Ph+ALL
provides a broad indication statement for Iclusig in resistant and intolerant
patients with Philadelphia positive leukaemias, including many patients who
have failed only one prior tyrosine kinase inhibitor,” stated Harvey J.
Berger, M.D., chairman and chief executive officer of ARIAD. “We appreciate
the substantial efforts of the CHMP in reaching this positive opinion so
rapidly as a consequence of Iclusig’s accelerated assessment designation, and
we look forward to the anticipated marketing authorization by the European
Commission around mid-2013.”
Although Iclusig was studied in Ph+ ALL patients who were resistant or
intolerant to dasatinib or nilotinib, nilotinib is not approved for the
treatment of Ph+ ALL.
The CHMP granted ARIAD accelerated assessment for its review of the Iclusig
marketing authorization application last summer. Accelerated assessment was
introduced by the European Union in 2005. Its aim is to help speed access for
patients to new medicines of major public-health interest. Companies can
request accelerated assessment provided they are able to demonstrate that
their product responds to an unmet medical need and is expected to have a
major impact on medical practice.
The CHMP is a scientific committee composed of representatives from the
27-member states of the European Union (EU), and Iceland and Norway. The CHMP
reviews medical product applications on their scientific and clinical merit
and provides advice to the European Commission, which has the authority to
approve medicines for the European Union.
CML is a cancer of the white blood cells that is diagnosed in approximately
7,000 patients each year in Europe^. CML and Ph+ ALL patients treated with
tyrosine kinase inhibitors (TKIs) can develop resistance or intolerance over
time to these therapies. Iclusig is a targeted cancer medicine discovered and
developed at ARIAD. It was designed by ARIAD scientists using ARIAD’s platform
of computational chemistry and structure-based drug design to inhibit BCR-ABL,
including drug-resistant mutants that arise during treatment. Iclusig is the
only TKI that has received a positive opinion from the CHMP for an indication
that includes CML and Ph+ ALL patients with the T315Imutation.
About CML and Ph+ ALL
CML is characterized by an excessive and unregulated production of white blood
cells by the bone marrow due to a genetic abnormality that produces the
BCR-ABL protein. After a chronic phase of production of too many white blood
cells, CML typically evolves to the more aggressive phases referred to as
accelerated phase and blast crisis. Ph+ ALL is a subtype of acute
lymphoblastic leukaemia that carries the Ph+ chromosome that produces BCR-ABL.
It has a more aggressive course than CML and is often treated with a
combination of chemotherapy and tyrosine kinase inhibitors. The BCR-ABL
protein is expressed in both of these diseases.
ARIAD Pharmaceuticals, Inc., headquartered in Cambridge, Massachusetts and
Lausanne, Switzerland, is an integrated global oncology company focused on
transforming the lives of cancer patients with breakthrough medicines. ARIAD
is working on new medicines to advance the treatment of various forms of
chronic and acute leukemia, lung cancer and other difficult-to-treat cancers.
ARIAD utilizes computational and structural approaches to design
small-molecule drugs that overcome resistance to existing cancer medicines.
For additional information, visit http://www.ariad.comor follow ARIAD on
This press release contains “forward-looking statements” including, but not
limited to, updates on regulatory developments in Europe. Forward-looking
statements are based on management's expectations and are subject to certain
factors, risks and uncertainties that may cause actual results, outcome of
events, timing and performance to differ materially from those expressed or
implied by such statements. These risks and uncertainties include, but are not
limited to, our ability to successfully launch, commercialize and generate
profits from sales of Iclusig; competition from alternative therapies, our
ability to obtain approval for Iclusig outside of the United States and in
additional indications; our reliance on third-party manufacturers, and on
specialty pharmacies for the distribution of Iclusig; preclinical data and
early-stage clinical data that may not be replicated in later-stage clinical
studies; the costs associated with our research, development, manufacturing
and other activities; the conduct and results of preclinical and clinical
studies of our product candidates; difficulties or delays in obtaining
regulatory approvals to market products; the adequacy of our capital resources
and the availability of additional funding; patent protection and third-party
intellectual property claims; our failure to comply with extensive regulatory
requirements; the occurrence of serious adverse events in patients being
treated with Iclusig or our product candidates; risks related to key
employees, markets, economic conditions, health care reform, prices and
reimbursement rates; and other risk factors detailed in the Company's public
filings with the U.S. Securities and Exchange Commission. The information
contained in this press release is believed to be current as of the date of
original issue. The Company does not intend to update any of the
forward-looking statements after the date of this document to conform these
statements to actual results or to changes in the Company's expectations,
except as required by law.
1. Rohrbacher M, Hasford J. Epidemiology of chronic myeloid leukaemia (CML).
Best Pract Res Clin Haematol. 2009 Sep;22(3):295-302. Based on current
estimate of population of Europe (738,199,000 in 2010).
ARIAD Pharmaceuticals, Inc.
Kendra Adams, 617-503-7028
For U.S. Media
ARIAD Pharmaceuticals, Inc.
Liza Heapes, 617-621-2315
For EU Media
Heather Grant, +44 (0) 207 632 1873
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