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Zavesca (Type C)

Currently, there are no FDA-approved therapies for Niemann-Pick Disease, type C in the United States, though a recent controlled study and a series of case reports suggest limited efficacy for miglustat, an inhibitor of glycosphingolipid biosynthesis.

Zavesca© (Miglustat), a medication developed by Actelion Pharmaceuticals, has NOT been approved by the U.S. Food and Drug Administration as a treatment for NPC for patients in the United States. But Zavesca© is an FDA approved medication of Gaucher’s disease, another Lisosomal storage disease. NPC Patients in the United States have gained access to a prescription for Zavesca when the patient’s treating physician has garnered the appropriate authorizations to prescribe.  For more information visit www.zavesca.com.

We have provided a table below with related articles showing how it has proven an affective treatment for Niemann-Pick Disease type C.

Publication Date Publication Name Article Title Article Author Article Summary
August 2,
2007
The Lancet Miglustat for treatment of NPC disease: a randomised controlled study Marc C Patterson, Darleen Vecchio, Helena Prady, Larry Abel, James E Wraith Establishing the effects of miglustat on several markers of NPC severity.
January,
2009
Journal of Child Neurology Long-Term Miglustat Therapy in Children with NPD Type C Marc C. Patterson, MD, FRACP, Darleen Vecchio, MS, Elizabeth Jacklin, RGN,
Larry Abel, PhD, Harbajan Chadha-Boreham, MSc, PhD, Cecile Luzy, MSc,
Ruben Giorgino, MD, DPhil, and James E. Wraith, MB, ChB, FRCPCH
Reporting on the open-label extension to the pediatric study of Miglustat on juveniles and adults with NPC.
August 4,
2009
Elesevier ~ Molecular Genetics and Metabolism Miglustat in patients with Niemann-Pick disease Type C (NP-C): A multicenter
observational retrospective cohort study
M. Pineda, J.E. Wraith, E. Mengel. F. Sedel, W.-L. Hwue, M. Rohrbach, B. Bembi, M. Walterfang,
G.C. Korenke, T. Marquardt, C. Luzy, R. Giorgino, M.C. Patterson
A report of findings from a retrospective observational cohort study assessing the effects of
miglustat on neurological disease progression in patients treated in the clinical practice setting.
December 30, 2009 Elesevier ~ Molecular Genetics and Metabolism Miglustat in adult and juvenile patients with Niemann–Pick disease type C:
Long-term data from a clinical trial
James E. Wraith, Darleen Vecchio, Elizabeth Jacklin, Larry Abel, Harbajan Chadha-Boreham,
Cécile Luzy, Ruben Giorgino, Marc C. Patterson
A report of data from a non-controlled, open-label extension to this initial randomized trial.
May 8th, 2012 Elesevier ~ Molecular Genetics and Metabolism Recommendations for the diagnosis and management of Niemann–Pick
disease type C: An update
Marc C. Patterson, Christian J. Hendriksz, Mark Walterfang, Frederic Sedel,
Marie T. Vanier, Frits Wijburg
on behalf of the NP-C Guidelines Working Group 1
A report on points of consensus among experts in the diagnosis and treatment of NP-C based on a follow-up
meeting in Paris, France in September 2011.

Insurance Appeal Letter & Documentation for Zavesca© (Miglustat)

NPC Patients in the United States have gained access to a prescription for Zavesca when the patient’s treating physician has garnered the appropriate authorizations to prescribe.  For more information visit www.zavesca.com.

Below are links to related articles and NNPDF families methods of obtaining Zavesca.
Harry Koujaian, whose daughter has NPC, has provided the community a template letter that can be used when appealing to your insurance company for compassionate use of Zavesca, along with further scientific documentation to help support the letter.   Many of the articles listed above are included here, with the relevant portions highlighted in yellow.

Jay Rosenthal also provided additional documentation that could be useful as well when appealing.