Search Orphan Drug Designations and Approvals
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Generic Name: | an antibody oligonucleotide conjugate (AOC), comprised of a human transferrin receptor 1 (TfR1) targeting, effector function null, humanized IgG1 antibody (AV01mAb) conjugated to one small interfering RNA (siRNA). |
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Date Designated: | 07/21/2021 |
Orphan Designation: | Treatment of Myotonic Dystrophy Type 1 (DM1) |
Orphan Designation Status: | Designated |
FDA Orphan Approval Status: | Not FDA Approved for Orphan Indication |
Sponsor: |
Avidity Biosciences 10975 N. Torrey Pines Road, Ste. 150 La Jolla, California 92037 United States The sponsor address listed is the last reported by the sponsor to OOPD. |
*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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