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Huntington’s Disease: Auspex Pharmaceuticals Gets FDA Orphan Drug Designation

November 7, 2014

Auspex Pharmaceuticals, a La Jolla, California-based biopharmaceutical company focusing on developing and commercializing medicines for rare diseases, announces November 6th, that the FDA grants Orphan Drug Designation (ODD) for investigational compound SD-809 for Huntington’s Disease (HD).

The following are clinical trials for SD-809:

•   Phase III registration clinical trial (First-HD) that is evaluating the efficacy and safety for treatment of chorea (abnormal involuntary movements) associated with HD – “on track to announce topline data in December 2014”

•   Phase III open-label switch & long-term safety (ARC-HD).

The following are planned clinical trials for SD-809 for additional orphan hyperkinetic movement disorder indications:

•   Phase II/III for drug-induced tardive dyskinesia

•   Phase Ib for Tourette Syndrome.

FDA Orphan Drug Designation Database Record For Auspex Pharmaceuticals

Generic Name: d6-tetrabenazine, deutetrabenazine
Trade Name: n/a
Date Designated: 11-05-2014
Orphan Designation: Treatment of Huntington’s Disease
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: Auspex Pharmaceuticals, Inc. 3366 N. Torrey Pines Court Suite 225 La Jolla, CA 92037


This is the 4th FDA ODD for Huntington’s Disease in 2014:

2014 FDA ODDs For Huntington’s Disease

# Generic Name Sponsor Company ODD Date
1 2-[4-(1-Methyl-4-pyridin-4-yl-lH-pyrazol-3-yl)-phenoxymethyl]-quinoline succinic acid Pfizer 06.02.14
2 Carbenoxolone Oxalys Pharmaceuticals (Canada) 07.02.14
3 5,7-dichloro-2-methylaminomethyl-8-hydroxyquinolone hydrochloride Prana Biotechnology (Australia) 09.04.14
4 d6-tetrabenazine, deutetrabenazine Auspex Pharmaceuticals 11.05.14


Please Note: “Nessler Cylinders” by Panek (Own work) [GFDL or CC-BY-3.0 | via Wikimedia Commons.

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