Chase the Cure, Inc.
a 501c3 Nonprofit working to get treatments out of the labs and into this generation of NPC patients
There is no current FDA approved treatment for
Niemann Pick Type C
Miglustat/Zavesca
Why could this drug help?
Often NPC is described as a disease in which cholesterol accumulates in the cells, and it does. However lipids & specifically glycolipids accumulate in the nervous system causing damage. Zavesca is thought to treat glycosphingolipidosis by reducing its buildup.
Please click the photo to go directly to the Zavesca Website..but then get back here! We have much to discuss and well, we would truly appreciate a donation to help further research and treatment options for Niemann Pick Type C!
Zavesca® (miglustat) is approved in the European Union for the treatment of progressive neurological manifestations in adult patients and pediatric patients with Niemann-Pick type C disease (NP-C). Zavesca® is the first treatment to be approved for patients with Niemann-Pick type C disease, a very rare, invariably progressive and eventually fatal neurodegenerative genetic disorder affecting both children and adults.
Zavesca® (100 mg miglustat) is the first and only oral medication approved for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease, and it may only be used in those patients for whom enzyme replacement therapy is unsuitable.
Additional Details
This drug DOES NOT have FDA approval in the United States for treatment of NPC. It is however approved as a treatment for Gaucher Type 1 disease. A doctor can give an NPC patient what is known as an off-label prescription for this drug. Some insurance companies may not cover Zavesca and it is VERY expensive. If your physician feels it is right for you or your loved one and you need help working with insurance the NPC Community at large jumps in to help. See our NPC Family page to connect. At this time NPC automatically qualifies a patient for Medicaid. Most caregivers are reporting that Medicaid is covering the drug.
Need More Information?
CONTACT ACTELION
Actelion Pharmaceuticals US, Inc
5000 Shoreline Court, Suite 200
South San Francisco, CA 94080
USA
Phone 1-650-624-6900
Fax 1-650-589-1501
DEAR NPC COMMUNITY: In January 2015 many NPC patients in the United States received letters from their insurance companies informing them that this drug would no longer be covered. If you are in that position, or are struggling to get first time approval please reach out. The community has created template letters to help you prove efficiacy and medical necessity to your insurance carriers. Allow your fellow parents to get you through the beurocratic maze quickly!