Thursday, October 27, 2016

FDA Does Not Approve Actelion’s Zavesca in US – Distressing News For Niemann Pick Type C Community

March 9, 2010 by  
Filed under Featured Stories

Addi and Cassi's monthly dose of the drug Zavesca made by Actelion

Actelion, the makers of Zavesca, today put out a brief statement saying that the U.S. Food and Drug Administration did not approve Zavesca and wants more information from the company on treating Niemann Pick Type C, a rare and dementing neurological disease that afflicts our six year old identical twins, Addi and Cassi.

Niemann Pick Type C disease is caused by the inability of the body to process cholesterol at the cellular level and as a result severely destroys the brain and organs like the spleen and liver.

The FDA is asking Actelion for more pre-clinical and clinical information on Zavesca before it will approve the drug.  The drug has been approved in the European Union, South Korea, Brazil, Russia, Australia and Canada for adult and pediatric patients with Niemann Pick Type C disease.

I hope Actelion will continue to press forward and make the investment in Niemann Pick Type C disease after coming this far.

This is surely a setback for our entire community as many people have worked for years to push this drug forward and were hoping for FDA approval. Addi and Cassi have been taking Zavesca for two+ years and many NPC kids are showing moderate improvements. It would be devastating if we could not have our twins on this drug when data shows it provides a benefit.

This is a lesson learned for me. Now that I am pursuing Cyclodextrin as a treatment for Niemann Pick Type C disease, I need find out exactly what the FDA is looking for in the way of pre-clinical data. I want to make sure that the data we are gathering on Addi and Cassi’s today will be useful in the future.

I would like to understand what thee FDAs top five measurements of success are and what it takes to get a drug approved for an ultra rare disease that afflicts so few. I don’t want to get in front of an FDA panel five years from now only to be told we did not collect adequate pre-clincal and clinical data.

Article Source

Share this:
  • Print
  • Digg
  • Facebook
  • Google Bookmarks
  • Diigo
  • email
  • PDF
  • Twitter
  • Yahoo! Bookmarks


6 Responses to “FDA Does Not Approve Actelion’s Zavesca in US – Distressing News For Niemann Pick Type C Community”
  1. The vertical leap test may also be used-to determine your state of restoration prior to a weight training,
    rate, or even exercise session. In case you
    overtrain your system by doing an extreme level of heavy weight or
    - speed education, itself will be manifested by the exhaustion first in your performance in motions requiring
    highspeed. You do not observe it significantly, but this sort
    of fatigue will tend to show itself rapidly being a decrease
    in performance of the vertical bounce. If this happens ideally you will
    want to cut back somewhat on the level of your work out(s) to recoup.

    Something you can certainly do is utilize
    your straight jump of howmuch quantity and power you should utilize to get a work out as being a barometer.

Speak Your Mind

Tell us what you're thinking...
and oh, if you want a pic to show with your comment, go get a gravatar!

CommentLuv Enabled