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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: cholest-4-en-3-one, oxime
Date Designated: 02/17/2009
Orphan Designation: Treatment of spinal muscular atrophy.
Orphan Designation Status: Designated/Designation Withdrawn or Revoked
Date Designation Withdrawn or Revoked: 03/04/2019
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Genentech, Inc.
A Roche Group Member
1 DNA Way
South San Francisco, California 94080
United States

The sponsor address listed is the last reported by the sponsor to OOPD.
*Exclusivity Protected Indications are shown for approvals from 01/01/2013 to the present.
*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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