In the "Emerald" studies of dialysis patients, the heart-safety rate was balanced between peginesatide and the other ESAs. (It actually trended slightly in favor of peginesatide.)
As a result of these divergent analyses of peginesatide's safety, Affymax and Takeda decided to ditch the non-dialysis treatment indication and only seek FDA approval for the treatment of anemia in dialysis patients.
The controversy or unknowns for next week's advisory committee meeting is how the FDA and the panel's experts view peginesatide's safety given the concerns raised by the non-dialysis patient data. Don't for a second believe that the FDA or the panel will ignore the worrisome "Pearl" data just because Affymax decided not to pursue approval in non-dialysis patients. I expect we'll hear a lot about these data, and it could derail the drug entirely.
Peginesatide's efficacy data in the "Emerald" dialysis patient population wasn't entirely squeaky clean either. The proportion of patients who were able to maintain hemoglobin levels within the target range -- a key secondary endpoint of the studies -- was statistically lower for peginesatide (63%) versus the other ESAs (71.7%).These mixed data set up what should be an interesting FDA advisory panel next Wednesday. I expect a lot of questions and discussion about peginesatide's overall safety profile and a deeper inspection of why the drug appears to be safer in dialysis patients. That's a bit counterintuitive since dialysis patients are sicker than non-dialysis patients. It's also helpful to view peginesatide in the broader context of FDA's years-long crackdown on the overuse and safety of ESAs. Is there a real unmet medical need for another ESA, even one that can be dosed more conveniently? Does once-monthly dosing even matter for dialysis patients, who require more frequent treatment, not less? Do the peginesatide studies raise more questions about the relationship between the way ESAs work and heart safety than they answer? Stay tuned for next week's FDA panel, and please tune into my live blog. RKN56 asks, "With Onyx Pharmaceuticals (ONXX) now in play, who are likely suitors and when do you think a deal gets done?" People have been speculating about Onyx's inevitable acquisition ever since Nexavar was first approved for kidney cancer in December 2005, so if it does happen, we can all scream "FINALLY!" Biomarin Pharmaceuticals (BMRN) is the only other drug company that comes to mind that's been the subject of equal or more takeout chatter over the years.
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