2 New FDA Orphan Drug Designations: Glioblastoma Multiforme And Duchenne Muscular Dystrophy

This Blog Post discusses two new FDA Orphan Drug Designations (ODDs) for the following two indications:

•   Glioblastoma Multiforme

•   Duchenne Muscular Dystrophy (DMD).

I – Blexion Pharmaceuticals And Glioblastoma Multiforme

Bexion Pharmaceuticals,  a Kentucky-based biotechnology company, focuses on the development of treatments for cancer. Initial products are based on a proprietary platform technology licensed from Cincinnati Children’s Hospital Medical Center.

Bexion Pharmaceuticals receives FDA Orphan Drug Designation (ODD) for Saposin C, an active ingredient in its proprietary drug BXQ-350 for Glioblastoma Multiforme on February 3rd :

Generic Name:	Saposin C
Trade Name:	n/a
Date Designated:	02-03-2015
Orphan Designation:	Glioblastoma Multiforme
Orphan Designation Status:	Designated
FDA Orphan Approval Status:	Not FDA Approved for Orphan Indication
Sponsor:	Bexion Pharmaceuticals, LLC 632 Russell St. Covington, KY

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Gliomas are tumors that arise from glial cells and can occur in the spinal cord or brain (most common location). Gliomas are the most common type of brain tumor. Glioblastoma Multiforme (GBM) is the most common glioma to occur in adults, diagnosed at an average age of 55. Gliomas usually have a poor prognosis, with survival rates of about 30% at 1 year and 14% at 2 years. There is currently no approved drug to improve survival.

“In pre-clinical studies, Bexion’s first-in-class biologic, BXQ-350 has shown promising results in selectively inducing cell death in the laboratory. BXQ-350 is a proprietary nanovesicle formulation of Saposin C (sphingolipid activator protein C, or SapC) and the phospholipid dioleoylphosphatidylserine (DOPS).”

II – Bristol-Myers Squibb And DMD

Bristol-Myers Squibb receives FDA ODD for the rare disease, DMD:

Generic Name:	Bivalent anti-human myostatin adnectin-IgG1
Trade Name:	n/a
Date Designated:	02-11-2015
Orphan Designation:	Duchenne muscular dystrophy
Orphan Designation Status:	Designated
FDA Orphan Approval Status:	Not FDA Approved for Orphan Indication
Sponsor:	Bristol-Myers Squibb Co. 5 Research Parkway Wallingford, CT 06492

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Please Note: “Chemicals in Flasks” by Joe Sullivan (Flickr) [CC-BY-2.0] | Wikimedia Commons.

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