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Orphan Drugs: Pfizer Receives FDA Designation For Huntington’s Disease

June 3, 2014









On June 2, the FDA grants Pfizer an Orphan Drug Designation (ODD) for PF-02545920 for Huntington’s Disease:


Generic Name: 2-[4-(1-Methyl-4-pyridin-4-yl-lH-pyrazol-3-yl)-phenoxymethyl]-quinoline succinic acid
Trade Name: n/a
Date Designated: 06-02-2014
Orphan Designation: Treatment of Huntington’s disease
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: Pfizer Inc. 445 Eastern Point Road Groton, CT 06340 The sponsor address listed is the last reported by the sponsor to OOPD.


PF-02545920 is a PDE10 Inhibitor that is in development at Pfizer for Huntington’s Disease and as an adjunctive treatment for Schizophrenia (reference Pfizer Pipeline as of 05/08/14). Pfizer is currently recruiting participants for a Phase II, double-blind randomized, sequential treatment group, placebo-controlled trial, to study the safety, tolerability, and brain function of 2 doses of PF-02545920 in patients with early Huntington’s Disease. Pfizer is working with the CHDI Foundation, a privately-funded, not-for-profit biomedical research organization dedicated to Huntington’s Disease, to evaluate PF-02545920.

For additional information, please reference the video from CHDI’s 8th Annual HD Therapeutics Conference (April 2013) for the presentation by Margaret M. Zaleska, PhD (Pfizer), titled, “Advancing Phosphodiesterase 10A (PDE10A) Inhibitor from Bench to Clinic”.

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

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