Iclusig ponatinib regulatory update

Germany's Federal Joint Committee (G-BA) said in a final benefit assessment that Orphan drug Iclusig ponatinib from Ariad has an "unquantifiable" additional benefit to treat chronic myelogenous leukemia (CML) and Philadelphia-chromosome positive (Ph+) acute lymphoblastic leukemia (ALL). G-BA said that because of the short follow-up for study data included in the dossier for Iclusig, lack of control data and inadequate historical control data, a quantitative assessment of the degree of Iclusig's effect and a quantification of the drug's additional benefit was "not possible." The European Commission approved the pan- BCR-ABL tyrosine kinase inhibitor (TKI) in July. It is indicated to treat chronic, accelerated or blast phase CML in patients 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 in patients who have the T315I variant of BCR-ABL tyrosine kinase; and for Ph+ ALL patients who are resistant to or intolerant of dasatinib and for whom imatinib is not appropriate, or in patients who have the T315I variant (see BioCentury, July 15, 2013).

Under drug pricing law AMNOG, the additional benefit of Orphan products is regarded as having been demonstrated by marketing authorization, though G-BA still determines the extent of the additional benefit. Ariad will now negotiate a price with Germany's Statutory Health Insurance Funds Association (GKV-Spitzenverband). Earlier this month, Ariad relaunched Iclusig in the U.S. after FDA approved a REMS and a revised label limiting use of the leukemia drug. The company had suspended U.S. marketing and distribution of the drug in late October at FDA's request due to the risk of arterial thrombotic events, which were observed in patients treated with the drug (see BioCentury, Jan. 20). ...