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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: synthetic DNA that modulates U1 small nuclear ribonucleoprotein misassembly
Date Designated: 03/13/2026
Orphan Designation: treatment of malignant glioma
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Aptah Biosciences
930 Brittan Avenue, MBC BioLabs
San Carlos, California 94070
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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