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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: levacetylleucine
Trade Name: Aqneursa
Date Designated: 09/08/2021
Orphan Designation: Treatment of Niemann-Pick Disease, Type C
Orphan Designation Status: Designated/Approved
IntraBio Inc.
270 Devon Road
Tenafly, New Jersey 07670
United States

The sponsor address listed is the last reported by the sponsor to OOPD.

Marketing approved:

1 Generic Name: levacetylleucine
Trade Name: Aqneursa
Marketing Approval Date: 09/24/2024
Approved Labeled Indication: treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing greater than or equal to 15 kg
Exclusivity End Date: 09/24/2031 
Exclusivity Protected Indication* :  treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing greater than or equal to 15 kg
*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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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