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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: RNA interference (RNAi) therapeutic, that targets the expression of TTR
Date Designated: 02/04/2025
Orphan Designation: treatment of transthyretin (TTR) -mediated amyloidosis (ATTR amyloidosis)
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Alnylam Pharmaceuticals, Inc.
300 Third Street
Cambridge, Massachusetts 02142
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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