Hadley Hope - Raising Awareness of Niemann- Pick Type C Disease


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FDA Denies Zavesca as approved drug for NPC

As most of you have been following, there is only one drug available for the treatment of NPC, called Zavesca.  It does not cure the disease, but it helps slow the progress of the disease, which helps families buy time, until a cure is found.  The problem is, this drug is available "off label", and is not yet approved by the FDA.  That means that only about 50% of the families are able to get the drug through their insurance company.

This year, the makers of Zavesca, Actelion, applied to the FDA to approve the drug in the U.S., which would allow all families access to the drug.  Zavesca is already approved in Europe, Russia, Canada, Japan, and other countries.  Last Month, in a prelimenary hearing, an advisory panel recommended to the FDA to approve Zavesca, based on the data of researchers and testimony of the families.

Tragically, we learned last week that the FDA has DENIED the application of Zavesca as an approved drug.  They are requesting more data, which will take years to get, and almost impossible to satisfy the high standards of the FDA.  The problem is: Niemann Pick Type C only has about 500 patients worldwide, of which very few are able to participate in a trial.  And, there are no concrete "biomarkers" availabe to test efficacy, only observational data.  Because there is not enough patients, "enough" will never be enough for the FDA.

Currently, we are fortunate that our insurance is paying the yearly $100K+ cost of Zavesca (reviewed yearly).  Unfortunately, we have spoken to many families that are not covered and were pinning their hopes on the FDA approval.  We had very high hopes that the FDA would do the right thing, but unfortunately that did not happen.  Please continue to pray that some day soon they will change thier hearts and minds.  We will continue to fight, and help in any way we can.





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