Archive for the ‘Drug Trials’ Category

No Bueno – FDA says hold off on Zavesca Approval

A day late and a dollar short… maybe. No approval from the FDA took place this week. In other countries like the European Union, South Korea, Brazil, Russia, Australia and Canada, Zavesca has been approved for use in adult and pediatric patients suffering from this fatal disease called Niemann-Pick Type C.

Our understanding of the news that broke out a few days ago was that they want more testing done from pre-clinical and clinical studies to develop additional information.

Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion commented: “We remain committed to bringing an approved treatment to patients suffering from NP-C disease and in this spirit we will continue the dialogue with the FDA.”

Zavesca-miglustat 100mgIt appears that Actelion is committed to finding the additional information needed to show the FDA that this could be one of the first FDA approved treatments for Niemann-Pick Type C.  Although this doesn't mean they will be cured, it could give someone like Brisan and Parker a few “extra” days living with NPC. As parents affected by this disease we are thankful that we currently have the opportunity to be apart of it even though Brisan and Parker aren't taking it. They will resume sometime in May this year.

That presents a different challenge for our family because we currently are on COBRA. It will be interesting what 12 months from now will look like especially if it isn't FDA approved because our future insurer might say H-E-double hockey sticks NO!

Here is a link to one of many articles: 


Round 2: N-Acetyl Cysteine & Biomarker Validation for Niemann-Pick Type C Drug Trial

Ding ding, round 2! Brisan and Parker started this week the 2 phrase of the N-Acetyl Cysteine (NAC) Drug Trial at the NIH. For the first week they have to ramp up on the drug again. Parker is at 9.5 ml x3 and Brisan is at 10ml x3. On the first phase it was 36ml x3 for Parker and 40ml x3 for Brisan.

As I posted originally on, a diagram that I used from NNPDF's site to show how this crossover study is taking place. You can read more about it here: NAC Drug Trial. Essentially they are looking how they can reduce oxidative stress on the cells. The main issue they can't fix yet but if they can find little things to help the “side effects”, then that is  a very small win overall for NPC. We haven't seen any major changes in how they act. If there is they are very minor that they aren't completely noticeable.

NAC trial timeline diagram

Overall the drug is pretty safe. You can actually purchase it from any pharmacy. It doesn't taste bad either! We view this as an opportunity to help in the research of Niemann-Pick Type C!

N-Acetyl Cysteine Drug Trial 900mg at the NIH.