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In September 2015, there are more than 25 potential therapies in the drug development pipeline for NPC, and some are so close to potential FDA approval.

Last updated: September 09, 2015
Basic Science Research Ideas Preclinical: Discovery IND Clinical Development NDA FDA Approval
Identification Optimization Safety & Manufacturing Phase 1 Phase 2 Phase 3
Vtesse – VTS-270 (Cyclodextrin)
Launched a global, multi-site Phase II/III clinical trial in September 2015.
NIH – Miglustat
Failed to get FDA Approval as a drug for NPC.
NIH – Vorinostat
Currently in a Phase I trial. (Already FDA approved as a drug for cancer.)
Orphazyme – Arimoclomol (HSP-70)
Observational clinical trial to start by March 2016.
CTD – Trappsol Cyclo (Cyclodextrin)
Future clinical trial in the works; will donate $1 million of Trappsol Cyclo for compassionate use.
Gene Therapy
Found to have no effect for NPC.
Anti-inflammatory (NSAID)
Verified to have benefit in NPC Mice (2007).
c-ABL Kinase
CDK Inhibitors
Cerebroside Synthase Inhibitors
Chemical Chaperones
Coenzyme Q9
Coenzyme Q10
Available as an over-the-counter supplement.
Enzyme Replacement Therapy
Already FDA approved as a drug for multiple sclerosis (MS).
Folding Regulators
Glucosylceramide Synthase Inhibitors
Iron Therapy
Metal-based Therapy
Already FDA approved as a drug for cancer.
Nuclear Orphan Receptors
Rab Overexpression
Small Molecules
Found to have no effect for NPC.
Stem Cell Therapy
Zinc Therapy
IND = Investigational New Drug NDA = New Drug Application

Last updated: September 09, 2015


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