FDA Sets April 24, 2012 as PDUFA Goal Date for Cell Therapeutics, Inc. (CTIC)' Resubmitted Pixantrone New Drug Application
12/6/2011 6:53:29 AM
SEATTLE, Dec. 6, 2011 /PRNewswire/ -- Cell Therapeutics, Inc. ("CTI") (NASDAQ and MTA: CTIC) today announced that the U.S. Food and Drug Administration's ("FDA") Division of Oncology Products 1 ("DOP1") has notified CTI that CTI's October 2011 resubmitted New Drug Application ("NDA") is considered a complete, Class 2 response to the FDA's April 2010 complete response letter ("CRL"). The FDA has set a Prescription Drug User Fee Act ("PDUFA") goal date of April 24, 2012 for a decision on the NDA. The NDA seeks accelerated approval of pixantrone to treat relapsed or refractory aggressive non-Hodgkin's lymphoma ("NHL") in patients who failed two or more lines of prior therapy.
Pixantrone is also under review for marketing approval in Europe with a Committee for Human Medicinal Products ("CHMP") opinion on the application tentatively scheduled on January 19, 2012.
"We look forward to working with the FDA and the European Medicines Agency ("EMA") to bring this drug to patients for whom there are currently no approved therapies," James A. Bianco, M.D. Chief Executive Officer stated.
CTI appealed the CRL for the pixantrone NDA based on the results of the PIX301 clinical trial. The FDA's Office of New Drugs ("OND") responded to CTI's appeal by providing CTI with an opportunity to resubmit the NDA with additional information, but without conducting an additional clinical trial. In June 2011, CTI met with the DOP1 to review CTI's plans for responding to the items noted in the CRL including CTI's plans for addressing the items noted by the OND in response to the appeal.
Pixantrone is a novel aza-anthracenedione that has distinct structural and physio-chemical properties that make its anti-tumor activity unique in this class of agents. Similar to anthracyclines, pixantrone inhibits Topo-isomerase II but unlike anthracyclines -- rather than intercalation with DNA -- pixantrone alkylates DNA -- forming stable DNA adducts with particular specificity for CpG-rich, hyper-methylated sites. These structural differences resulted in significantly enhanced anti-lymphoma activity compared to doxorubicin in preclinical models. In addition, the structural motifs on anthracycline-like agents that are responsible for the generation of oxygen free radicals and the formation of toxic drug-metal complexes have also been modified in pixantrone in an effort to prevent the binding of iron and perpetuation of superoxide production -- both of which are the putative mechanism for anthracycline induced acute cardiotoxicity. These novel pharmacologic differences may allow re-introduction of anthracycline-like potency in the treatment of relapsed/refractory diffuse large lymphoma without unacceptable rates of cardiotoxicity.
About Cell Therapeutics, Inc.
Headquartered in Seattle, CTI is a biopharmaceutical company committed to developing an integrated portfolio of oncology products aimed at making cancer more treatable. For additional information, please visit www.CellTherapeutics.comhttp://www.CellTherapeutics.com.
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This press release includes forward-looking statements that involve a number of risks and uncertainties the outcome of which could materially and/or adversely affect actual future results and the market price of CTI's securities. Specifically, the risks and uncertainties that could affect the development of pixantrone include risks associated with preclinical and clinical developments in the biopharmaceutical industry in general, and with pixantrone in particular, including, without limitation, the potential failure of pixantrone to prove safe and effective for the treatment of relapsed or refractory NHL and/or other tumors as determined by the FDA and/or the EMA, that accelerated approval by the FDA of pixantrone may not occur, that CTI may not be able to address satisfactorily the two key matters raised by the OND or other matters raised by the DOP1, the OND and/or the FDA, that CTI's interpretation of the guidance provided by the OND, the DOP1 and/or the FDA may be different than the intent of the OND, the DOP1 and/or the FDA, that the OND, the DOP1 and/or the FDA may change its guidance, that the PIX301 study may not be deemed successful, that upon a re-review and/or resubmission of the NDA the FDA may find pixantrone to not be safe and/or effective, that the PIX301 study may still be deemed to be a failed study, that the FDA may require an additional clinical trial of pixantrone, that if CTI conducts an additional clinical trial, it may not demonstrate the safety and effectiveness of pixantrone, that CTI may not be able to provide satisfactory information in response to the CRL, that the FDA may not complete the review of the NDA by the PDUFA goal date of April 24, 2012, that the CHMP may not render an opinion on CTI's application for pixantrone with the EMA by January 19, 2012, CTI's ability to continue to raise capital as needed to fund its operations, competitive factors, technological developments, costs of developing, producing and selling pixantrone, and the risk factors listed or described from time to time in CTI's filings with the Securities and Exchange Commission including, without limitation, CTI's most recent filings on Forms 10-K, 10-Q and 8-K. Except as may be required by law, CTI does not intend to update or alter its forward-looking statements whether as a result of new information, future events, or otherwise.
Lindsey Jesch Logan
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SOURCE Cell Therapeutics, Inc.