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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals

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Generic Name: recombinant human acid a-glucosidase co-administered with N-butyl-deoxynojirimycin (recombinant human acid a-glucosidase/miglustat)
Date Designated: 09/13/2017
Orphan Designation: Treatment of Pompe disease
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Amicus Therapeutics, Inc.
3675 Market Street
Philadelphia, Pennsylvania 19104
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.