ARIAD announces that Iclusig(TM) receives positive CHMP opinion for approval in the European Union

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[1]. 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.

Reference:

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).