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How Many FDA Orphan Drug Designations Does Prosensa Receive For Duchenne Muscular Dystropy ?

January 31, 2013

Prosensa, a Dutch biopharmaceutical company, announces on January 29, 2013, that it has achieved Orphan Drug Designation (ODD) in the United States for all of its compounds for the treatment of Duchenne Muscular Dystrophy (DMD).

Prosensa currently has 6 exon-skipping compounds in development for treating DMD. The FDA grants ODD to Prosensa’s 4 preclinical compounds PrO045, PRO052, PRO053, and PRO055 DMD on January 23, 2013. Prosensa’s 2 other Exon-specific compounds, PRO044 and PRO051 (Drisapersen), receive ODD prior to 2013.  PRO051 (Drisapersen), is being developed in collaboration with GlaxoSmithKline, in a fully enrolled, late stage Phase III clinical trial of 186 patients, in 47 trial sites in 20 countries. The chart below is generated from the FDA’s Orphan Drug Product Designation Database Application.

Generic Name Designation Date Indication
Exon 44 specific   phosphorothioate oligonucleotide 11.05.09 DMD
Exon 45 specific phosphorothioate   oligonucleotide 01.23.13 DMD
Exon 51 specific   phosphorothioate oligonucleotide 08.25.09 DMD
Exon 52 specific phosphorothioate   oligonucleotide 01.23.13 DMD
Exon 53 specific phosphorothioate   oligonucleotide 01.23.13 DMD
Exon 55 specific phosphorothioate   oligonucleotide 01.23.13 DMD

As noted in a previous December 2012 Orphan Druganaut Blog Post, Henri Termeer, former Genzyme CEO and Chairman who retired in 2011 after almost 30 years, joins Prosensa as a strategic adviser “providing advice for the biotech’s corporate strategy and growth plans’’. Mr. Termeer built Genzyme into a leader in rare diseases and orphan drugs.

The main competitor to Prosensa is Sarepta Therapeutics’ orphan drug Eteplirsen for DMD.

References

Prosensa Drug Pipeline

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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