Cell Therapeutics, Inc. ("CTI") (Nasdaq and MTA: CTIC) today announced that it intends to appeal the U.S. Food and Drug Administration's (the "FDA") previously disclosed decision regarding the pixantrone New Drug Application ("NDA") to treat patients with relapsed/refractory aggressive non-Hodgkin's lymphoma ("NHL"). CTI had requested accelerated approval of its NDA for this patient group for which there are no currently approved drugs. The FDA issued a Complete Response Letter to CTI related to this NDA stating, in part, that CTI should conduct an additional clinical trial prior to approval. CTI is preparing to file an appeal under the FDA's Formal Dispute Resolution process. CTI reached this decision by taking into account that CTI believes there are no approved or effective therapies for patients with relapsed or refractory aggressive NHL beyond second relapse, and that PIX 301 was the first and only randomized trial in this patient group to demonstrate significant improvement in clinically relevant endpoints including complete response rate, overall response rate, and progression free survival while being safe and effective in this indication.
"The PIX 301 study produced robust clinically meaningful results including improvement in response rate and progression free survival that could have a significant impact on treating the disease in this setting," said Dr. Stanley M. Marks, Director of Clinical Services and Chief Medical Officer for the University of Pittsburgh Cancer Centers and Chief of the Division of Hematology/Oncology at UPMC.
"After discussions with a number of leading U.S. and international lymphoma experts and leading biostatisticians, all of whom have reviewed the PIX 301 protocol, statistical plan and trial results, we felt encouraged to appeal the initial decision and have our data reviewed in the context of a trial that achieved statistically and clinically meaningful primary and secondary endpoints in this end stage patient population for whom there are no approved or effective agents," noted Jack Singer. M.D., Chief Medical Officer at CTI. "We are committed to conducting a confirmatory trial in this patient population but continue to strongly believe that the PIX 301 data met the requirements for accelerated approval with sufficient scientific rigor."
FDA regulations provide a Formal Dispute Resolution mechanism to obtain review of any FDA decision, including a decision not to approve an NDA, by raising the matter with the supervisor of the Office that made the decision. The Formal Dispute Resolution process exists to encourage open, prompt discussion of scientific (including medical) disputes and procedural (including administrative) disputes that arise during the drug development, new drug review, and post-marketing oversight processes.
CTI expects the FDA decision on the appeal in the fourth quarter of 2010.
CTI continues to prepare for the initiation of an additional pixantrone clinical study in the U.S. that would serve as either a post-approval confirmatory study or as a registration study for approval in the U.S. Also, CTI expects to file a Marketing Authorization Application ("MAA") for approval of pixantrone in Europe before the end of this year.
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