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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-based vector engineered to express full-length human ATPase Secretory Pathway Calcium Transporting 1 (ATP2C1)
Date Designated: 11/20/2025
Orphan Designation: treatment of Hailey-Hailey disease (HHD)
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Krystal Biotech, Inc.
2100 Wharton Street, Suite 701
Pittsburgh, Pennsylvania 15203
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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