Cell Therapeutics, Inc. (“CTI”) has filed a formal appeal with the Office of New Drugs in the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research to contest the decision from earlier this year on CTI’s New Drug Application (“NDA”) for pixantrone as a therapy for relapsed/refractory aggressive non-Hodgkin’s lymphoma (“NHL”). CTI had requested accelerated approval of its pixantrone NDA for a patient group as there are no drugs currently approved in this clinical setting.
A portion of the Office of Oncology Drug Products’ requested Complete Response Letter stated that CTI’s prior clinical trial, PIX301, did not demonstrate efficacy, and that CTI should conduct an additional clinical trial prior to approval. CTI is preparing an additional study, but still contests this statement on the grounds that, according to Richard Kay, PhD, Statistical Consultant, RK Statistics Ltd., Honorary Visiting Professor, School of Pharmacy, Cardiff University, “the FDA diverged from accepted statistical principles and practices when the FDA applied a more stringent statistical significance level in concluding that the PIX301 primary analysis required an adjustment for type 1 error as if an interim analysis had been conducted. This was not the case in the PIX301 trial where only a single final analysis was undertaken. We believe the results of the PIX301 clinical trial should be analyzed with the appropriate threshold that is standard for trials of this type in which only one efficacy analysis was conducted.”
This appeal will be brought forth for review to the supervisor of the FDA office that made the initial decision per FDA regulations. Based on the guidance documents available from the FDA, CTI expects an FDA decision on the appeal within the next four months.
CTI is already preparing to initiate another trial, PIX306, which would serve as either a post-approval confirmatory trial or a second registration trial for approval and has submitted documentation for a Special Protocol Assessment (“SPA”) to the FDA to review. Additionally, on November 17, 2010, the Company’s Marketing Authorization Application for pixantrone that was filed with the European Medicines Agency was accepted and is being reviewed.
More information on Cell Therapeutics, Inc. and its pipeline of drugs can be found on the company’s website at www.celltherapeutics.com.
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