Quick Links: Skip to main page content Skip to Search Skip to Topics Menu Skip to Common Links
  • Decrease font size
  • Return font size to normal
  • Increase font size
U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
  • Print
  • Share
  • E-mail
-
Generic Name: antisense oligonucleotide complementary to the exonic splicer enhancer sequence at intron 26 of the centrosomal protein 290 pre-mRNA
Date Designated: 05/25/2016
Orphan Designation: Treatment of Leber's congenital amaurosis.
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
ProQR Therapeutics IV B.V.
Zernikedreef 9
Leiden 2333 CK
Netherlands

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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.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