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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 Jan. 1, 2013, to the present.