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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals

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Generic Name: human cystic fibrosis transmembrane conductance regulator (hCFTR) messenger RNA (mRNA) complexed with branched polyethyleneimine (bPEI)
Date Designated: 11/03/2015
Orphan Designation: Treatment of patients with cystic fibrosis.
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
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200 Sidney Street
Suite 310
Cambridge, Massachusetts 02139
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.