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Research Grants Drug Development Pipeline

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
Allopregnanolone
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
Curcumin
Available as an over-the-counter supplement.
Enzyme Replacement Therapy
Fingolimod
Already FDA approved as a drug for multiple sclerosis (MS).
Folding Regulators
Glucosylceramide Synthase Inhibitors
HDAC
HSP25
Iron Therapy
Metal-based Therapy
Nilotinib
Already FDA approved as a drug for cancer.
Nuclear Orphan Receptors
Rab Overexpression
Small Molecules
SSRI
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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