Cell Therapeutics (CTIC) saw an ominous cloud pass over when the Office of New Drugs (OND) of the U.S. Food and Drug Administration (FDA) allowed it to re-submit the new drug application (NDA) for its lead pipeline candidate, pixantrone, for review without the need for an additional trial.
Pixantrone is being developed for the treatment of relapsed or refractory aggressive non-Hodgkin’s lymphoma (NHL) in patients who have not responded to other treatment options.
In April 2010, the FDA had issued a complete response letter (CRL) for pixantrone based on concerns that the clinical trials (PIX301) have not sufficiently proved the effectiveness of the drug. The FDA asked Cell Therapeutics to conduct an additional trial to determine the safety and efficacy of Pixantrone.
Although the company began an additional trial for pixantrone, in March 2011, Cell Therapeutics appealed to the OND in December to reverse the FDA’s decision to deny approval of pixantrone. Cell Therapeutics met with the FDA OND and presented documents supporting the efficacy of pixantrone for the desired indication.
Consequent to a thorough review of the appeal as well as the NDA, the OND informed Cell Therapeutics that though the PIX301 trial did not adequately demonstrate the efficacy of pixantrone, the conclusion of the CRL that PIX301 was a failure would also be incorrect.
Cell Therapeutics will not need to conduct an additional study to demonstrate the efficacy of pixantrone. However, it can re-file the NDA for review if two criteria are met. Cell Therapeutics will have to conduct an additional independent radiologic review of the PIX301 trial to determine the soundness of the primary endpoint.
Apart from that, it will have to submit new information regarding circumstances of stopping trial early. The satisfactory submission of the required data by Cell Therapeutics would thus render the PIX301 trial successful with its primary and secondary endpoint duly met.
Cell Therapeutics plans to arrange for a meeting with the appropriate division of the FDA's Office of Oncology Drug Products and submit the required information.
We are encouraged by OND’s decision and believe that it removes a major overhang for Cell Therapeutics by easing the uncertainty on the approval of its lead pipeline candidate.
Our Recommendation
We currently have a Neutral recommendation on Cell Therapeutics, which is supported by a Zacks #3 Rank tantamount to a short-term Hold rating.
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