Tuesday, February 24, 2015

FDA Approves Farydak (Panobinostat)

This is a victory for myeloma patients, and perhaps for all patients facing a life-threatening disease.

Farydak (panobinostat) is a new oral drug approved specifically for use with bortezomib (Velcade) and dexamethasone to treat patients who have previously undergone at least two prior regimens, including Velcade and an immunomodulatory agent (thalidomide, Revlimid, or Pomalyst).

It comes with this boxed warning:

In other words it can be very unpleasant and can put your life at risk.  Further, in addition to the symptoms listed in the box, it can cause a reduction in platelets, neutrophils, or red cells, and can cause other serious problems.

So why is this a victory for patients?  Because Farydak can extend the lives of patients taking Velcade by months, perhaps years, that's why.

Nevertheless, last November the FDA Oncologic Drugs Advisory Committee (ODAC) voted 5 to 2 against approval of Farydak, judging that the potential benefits did not outweigh the risks.  This is a panel of "experts" (not a patient among them) deciding by themselves what risks WE patients should be permitted to take, and attempting to block us from all access to this drug.  In a recent speech I said "for people in my shoes, the side effect of NOT having the drug is worse – we call it death"!  

All of the new FDA-approved drugs have a daunting list of possible side effects, but we take them anyway because we don't like the alternative.  And as a result the median survival for myeloma patients has doubled during the 12 years since my diagnosis.  In my own case the advertised side effects of my little magic pill have been mostly absent - I've run 58 marathons now in my seven years on Pomalyst.

I congratulate the FDA on their understanding of this issue and their willingness to let us, with our pretty damn smart doctors, make the life-determining decisions ourselves.  This seems new, and I hope it is a harbinger of things to come.

Farydak is new too.  It works on an entirely new principle - it's called a histone deacetylase (HDAC) inhibitor - with the potential to be combined with any of the drugs that work on other principles. There is much to be learned about Farydak.  I hope I never need it, but I'm glad to have it in my quiver.

For more about access to emerging treatment technologies, please visit Closing the Gap Now .

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