Lysosomal Storage Disorders: Gene Therapy And FDA Orphan Designations
No gene therapy has ever been approved for sale in the United States. In Europe, the EMA (European Medicines Agency), has given regulatory approval to UniQure, to sell its gene therapy, Glybera, for the treatment of the rare disease, Lipoprotein Lipase Deficiency (LPLD).
This is the twelfth Blog Post in a continuing series that examines Lysosomal Storage Disorders (LSDs) in the rare disease and orphan drug space. This Blog Post identifies gene therapies for LSDs that have received FDA Orphan Drug Designation in 2013 – 2014.
FDA Orphan Designated Gene Therapies For LSDs (2013 – 2014)
|Drug Name||Sponsor Company||Indication||FDA ODD Date|
|AAV-G6Pase vector||GlyGenix Therapeutics||Glycogen storage disease type Ia||03.11.13|
|Recombinant adenovirus vector AAV2/rh8 expressing human B-hexosaminidase A & B subunits||National Tay-Sachs & Allied Diseases Association (NTSAD)||Tay-Sachs Disease||03.25.13|
|Recombinant adeno- associated virus vector AAV2/rh8 expressing human B-hexosaminidase A and B subunits||National Tay-Sachs & Allied Diseases Association (NTASD)||Sandhoff Disease||03.25.13|
|Adeno associated viral vector serotype rh.10 carrying the human SGSH and SUMF1 cDNAs||Lysogene (France)||MPS III A (Sanfilippo Syndrome)||05.06.13|
|Recombinant AAV9 expressing human sulfoglucosamine sulfohydrolase||AbeonaTherapeutics||MPS III A (Sanfilippo Syndrome)||04.29.14|
|Recombinant AAV9 expressing human alpha-N-acetylglucosaminidase||Abeona Therapeutics||MPS III B (Sanfilippo Syndrome)||04.30.14|
Please Note: “DNA Repair” courtesy of Tom Ellenberger, Washington University School of Medicine in St. Louis. [Public domain] | Wikimedia Commons..
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