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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: an antibody-siRNA conjugate, comprised of a humanized anti-TfR1 IgG1 monoclonal antibody covalently linked via a bis-maleimide non-cleavable linker to a double-stranded small interfering RNA directed to DMPK (myotonic dystrophy protein kinase) mRNA
Date Designated: 10/17/2025
Orphan Designation: treatment of myotonic dystrophy type 1 (DM1)
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
ChainGen Biopharma Ltd.
2555 Xiupu Road, Bldg A11 2F
Shanghai
China

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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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