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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: messenger RNA encoding for adenine base editor protein and guide RNA targeting the angiopoietin like 3 (ANGPTL3) gene
Date Designated: 09/11/2026
Orphan Designation: treatment of homozygous familial hypercholesterolemia
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Verve Therapeutics, a Wholly Owned Subs of Eli Lilly and Co
201 Brookline Ave, Suite 601
Boston, Massachusetts 02215
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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