News about Nplate seems to focus on the fact that the FDA missed the review deadline by about a month.
Oy vey. The drug industry in general is apparently pretty crabby about the idea that the FDA may be getting a little bit more cautious about approving new drugs after the Vioxx debacle. Heck, when you spend an average of 15 years developing a drug, you don’t want it sitting on the shelf for an extra month or two—regardless of any potential for deadly side effects.
Amgen’s platelet-stimulating drug may well have sat there for a reason.
Of course the FDA had questions. 14 of the 204 patients studied apparently had adverse reactions, eight patients developed a resistance, and there’s an increased risk of clotting or of serious bleeding when the patient stops taking the drug. The FDA was also concerned about abnormal marrow growth and malignant tumors.
“Serious adverse reactions associated with Nplate in clinical studies were bone marrow reticulin deposition and worsening thrombocytopenia after Nplate discontinuation. Additional risks include Bone Marrow Fibrosis, Thrombotic/Thromboembolic Complications, Lack or Loss of Response to Nplate, and Hematological Malignancies and Progression of Malignancy in Patients with a Pre-existing Hematological Malignancy or Myelodysplastic Syndrome (MDS),” reads Amgen’s disclaimer.
So the FDA had to figure out whether Nplate should be approved, and if so, under what—if any—conditions. Which turned out to include an ongoing long-term safety study.
Everything has consequences. Anyone with a platelet disorder that hasn’t responded to other treatments may well decide that the risks are worth it, and Amgen is to be applauded for developing a last-ditch drug. But patients deserve to be informed about what those risks are, and the drug industry should back off on criticism of the FDA’s requests for more safety data or a lengthy pause for thought.
What's your opinion? If you take Nplate, are you having any side-effects, and if so, are they what you expected? Do you think the FDA took too long to approve Nplate?
Monday, January 5, 2009
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I have been on Nplate since Nov 08 as an alternative to splenectomy and other first line treatments. I am very concerned about the side effects as they have been moderate in my case. I have my good weeks and bad. Overall, it has not proven to be a good method for reducing the dependency on other treatments to raise platelets to the mandated 200 thousand level the Nexus program has in their instructions.
I have had 8 Nplate injections since my diagnosis on Valentine's Day. My doctor had me skip an injection 2 weeks ago, to see if my count would maintain, but it dropped from 154K to 108K. We resumed and my count jumped up to 220K. I am glad that they are back to a "safe" level, but am wondering how long I will have to be on this drug. My doctor hasn't ruled out an acute diagnosis, so I'm still holding out for that.
As far as side-effects are concerned, I guess I haven't been on the drug long enough to comment on any long-term effects, but I have experienced the intense headaches/migraines from time to time. This occurred more often when I first began the drug.
Courtney, please keep us updated. I hope that Nplate's working better for you now, but if not, let us know what IS working.
kcog25, have you found any good antidotes to the headaches/migraines? I wonder whether Nplate depletes certain nutrients in your body, the way statins deplete CoQ10. If so, there might be some variety of supplement that would complement Nplate--have you asked your doctor about that?
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