Brisan and Parker Stults | Living with Niemann-Pick Type C

Posts Tagged ‘Zavesca’

Last week was a very busy week. Brisan and Parker both started physical therapy at Children's Mercy Hospital. It is kinda of funny that they “started” when we went almost two years ago and told us that “they didn't know what they could help us on” and that “oh…Medicaid will pay for these for visits”. HA…that wasn't the case with Medicaid. The good part is they are getting additional therapy. That is what matters most.

Also they met with their new neurologist. They started the boys on a cataplexy drug this week as well which is one pill at night. It should help manage when they lose muscle control and fall. She also consulted with Dr. Marc Patterson on resuming the boys on Zavesca. So that will be started up again very soon! That stuff is pretty expensive (like 190,000 for both of them). It will be interesting when we will have to find new commercial insurance at the end of the year.

On Wednesday August 11th, Brisan and Parker had a minor surgery to remove the granulation tissue around their G-tubes. Starting next Wednesday they both will start school. For Brisan it will be Kindergarten! Big day for the big birthday boy this month. On the 27th he will be 6!  We still can't believe it!

Jennifer's Dad Tim purchased a swing set for the boys. Last Sunday we started putting that monster together! Now we know Brisan and Parker probably aren't going to be able to enjoy it like more traditional kids their age but hopefully they will find something they will enjoy. Duncan probably will! The neighbor girl was telling her mommy that “mommy they are building my swing set”. HA

Parker and Brisan are doing better. Parker seems to be walking in an improved state.  Wednesday Duncan decided to jump on the couch and broke Jennifer's nose. She isn't feeling well and has an appointment with the ENT on Monday to see what can be done to resolve the issue. She posted the x-rays on her Facebook profile page.

April 21-24 we will have our last visit to the NIH for the NAC drug trial. That Thursday will be used to provide insight into Brisan's sinuses and polyps along with the finishing touches of paperwork / examinations they will perform. Nothing major expect 14 tubes of blood! In May we will start Zavesca back up but I am sure we will talk with Dr. Porter more about it to get his insight.

Brisan and Parker's port-a-cath sites are looking good. Monday will be the first time they can access Parker's site for his infusions. They also took Parker off AMICAR and gave him another drug in its place.

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.”

It 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: 

• Actelion receives FDA complete response letter for Zavesca (miglustat) for the treatment of Niemann-Pick type C disease

• FDA Does Not Approve Actelion’s Zavesca in US

• Actelion receives FDA complete response letter for Zavesca