FDA Panel Rules vs. Pixantrone – Analyst Blog

In a major disappointment for Cell Therapeutics’ (CTIC), the US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee unanimously decided against the approval of the company’s lead candidate pixantrone, citing lack of sufficient clinical data.

 

Cell Therapeutics is seeking approval of pixantrone for the treatment of relapsed or refractory aggressive non-Hodgkin’s Lymphoma (NHL) in patients who have not responded to other treatment options.

 

Although the FDA is not bound to follow the panel’s decision, it generally does so. It is quite unlikely that the agency would approve a drug that has been voted against unanimously. A final decision on pixantrone should be taken by April 23.

 

We were concerned about pixantrone’s approval due to certain issues raised by the FDA reviewers related to the study design. 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.

 

In July 2009, Cell Therapeutics initiated the process of obtaining marketing approval for pixantrone in Europe and received orphan drug status from the European Medicines Agency. However, the possibility of approval is remote, given the setback in the US.

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 on an uptrend as the company has been preparing for the potential launch of pixantrone. Expenses are going to soar if the FDA asks the company to conduct additional trials. We have a Neutral recommendation on the stock.