Serious adverse events reported in the study overall, according to Celgene, included 12 patients with pneumonia, eight patients with neutropenia and six patients with thrombocytopenia. Another six patients suffered from neutropenia with fever, a very serious sign of infection that forces sufferers to be hospitalized.
But while Celgene said that the other 12 deaths in the study were not related to Revlimid, company executives acknowledged that some of these deaths were also caused by pneumonia (they wouldn't disclose an exact number). Given that Revlimid lowers blood counts and makes patients susceptible to serious infection, Celgene critics question how only two deaths are being tied to the drug. If more of the patient deaths resulted from serious infections, Revlimid's toxicity was at least partially responsible, critics charge.
The question over how many patients in the study died because of Revlimid is further muddled by the fact that Celgene and a few of its investigators, including List, were charged with characterizing the patient deaths. There was no external and/or independent panel of doctors used to determine the cause of the patient deaths.
The best way to measure the efficacy and safety of any drug is to conduct a large, randomized and controlled study in which some patients receive the drug and others receive a placebo. However, this is something that Celgene chose not to do with Revlimid in these low-grade, 5 q minus MDS patients.
As recently as last week, at an advisory panel meeting held to review two cancer drugs, FDA officials reiterated their strong preference for controlled drug studies and expressed frustration that an increasing number of drug companies are seeking drug approvals based on uncontrolled studies.
Sometime next year, it will be the FDA that will ultimately decide whether Revlimid is an approvable drug for MDS patients. Until then, Celgene bulls and bears will stick to their guns, which could have the effect of keeping Celgene's stock price in a fairly tight trading range.
Safety data were priority No. 1 Sunday night, but Celgene also presented strong efficacy data: Patients enrolling in this study required regular blood transfusions to keep their MDS under control. But after taking Revlimid, approximately 64% of patients no longer required blood transfusions.
A final thought: Celgene is also developing Revlimid as a treatment for multiple myeloma, a cancer affecting plasma cells in the bone marrow. One fund manager at the confab here said the Revlimid-MDS dust-up is just noise compared with the big commercial opportunity if/when the drug is approved for multiple myeloma. That's why he said he owns Celgene and is willing to weather any short-term volatility to see it through. Celgene is running phase III trials of Revlimid in multiple myeloma, with data expected next year.