In a major setback, Cell Therapeutics (CTIC) has received a complete response letter (CRL) from the US Food and Drug Administration (FDA) for Pixuvri (pixantrone dimaleate). The company has been seeking approval for pixantrone for the treatment of relapsed or refractory aggressive non-Hodgkin’s lymphoma (NHL) in patients who have not responded to other treatment options.
However, this decision was expected as last month the FDA’s Oncologic Drugs Advisory Committee had unanimously decided against the approval of the candidate, citing the lack of sufficient clinical data. Some issues were raised by the FDA related to the study design at the advisory committee meeting. The same concerns led the FDA to take this decision.
The study that opened in June 2004 sought to enroll 320 patients. However, the enrollment was challenging and was stopped in March 2008 after enrolling 140 patients, less than half the original plan. The FDA has asked Cell Therapeutics to conduct an additional trial to determine the safety and efficacy of pixantrone.
Cell Therapeutics will request a meeting with the FDA to decide the design of the follow-on trial. Meanwhile, the company has decided to pursue expanded access program for pixantrone while it conducts an additional study.
Cell Therapeutics is also looking at obtaining marketing approval for pixantrone in Europe where the drug has received orphan drug status. However, the possibility of approval there, too, is remote, given the regulatory hurdle witnessed in the US. The company is meeting its clinical experts for their feedback and guidance for the marketing application, which is expected to be submitted in the third quarter of 2010.
Cell Therapeutics was banking heavily on the approval of pixantrone. The company does not have any marketed product at present; it derives revenues primarily from licenses and contracts.
We are also concerned about the company’s liquidity position. Operating expenses are likely to increase with the need for additional trials. We have a Neutral recommendation on the stock.