Iclusig ponatinib regulatory update

FDA granted accelerated approval to Iclusig ponatinib from Ariad to treat chronic myelogenous leukemia (CML) and Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) that is resistant or intolerant to prior treatment with tyrosine kinase inhibitors (TKIs). The approval comes more than 3 months ahead of the March 27, 2013, PDUFA date. Iclusig's label includes a black box warning about the risk of arterial thrombosis and liver toxicity observed with use of Iclusig. On a conference call to discuss the approval, the company said it does not believe the warning will affect the drug's uptake. Ariad plans to launch the drug in the U.S. in about 2 weeks with a wholesale acquisition cost (WAC) of $9,580 for a month's supply, or about $115,000 per year.

The approval was based on data from the pivotal Phase II PACE trial, which did not include a control arm and was conducted in a heavily pre-treated patient population. For full approval, Ariad is required to submit 2-year follow-up data from PACE, as well as data from the ongoing controlled Phase III EPIC trial in newly diagnosed patients. On the conference call, the company said it believes the EPIC data will provide "a good feel" for the actual frequency of adverse events and whether they are Iclusig-related. ...