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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: messanger ribonucleic acid (mRNA)-based therapeutic agent encoding human methylmalonyl-coenzyme A mutase
Date Designated: 03/07/2018
Orphan Designation: Treatment of isolated methylmalonic academia (MMA) due to methylmalonyl-Coenzyme A mutase (MUT) deficiency
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Moderna Therapeutics, Inc.
200 Technology Square
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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