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Duchenne Muscular Dystrophy: 2013 FDA Orphan Drug Designations

December 14, 2013

Since 2012, Duchenne Muscular Dystrophy (DMD) has attracted interest from both investors and the media. This is the first Blog Post in a series over the next week that will examine DMD in the rare disease and orphan drug space.

This Blog Post reviews the eight 2013 FDA Orphan Drug Designations (ODDs) for the treatment of DMD that are granted in 2013. Please note that Sarepta Therapeutics’ Eteplirsen receives FDA ODD in 2007 and Prosensa/GSK’s Drisapersen receives FDA ODD in 2009.

2013 FDA Orphan Drug Designations For DMD

Row Num

Generic Name Designation Date Orphan Designation


O-(3-piperidino-2-hydroxyl-1-propyl)-nicotinic acid   amidoxime hydrochloride 01-15-2013 DMD


Exon 45 specific phosphorothioate oligonucleotide 01-23-2013 DMD


Exon 52 specific phosphorothiate oligonucleotide 01-23-2013 DMD


exon 53 specific phosphorothioate oligonucleotide 01-23-2013 DMD


Exon 55 specific phosphorothioate oligonucleotide 01-23-2013 DMD


givinostat 04-12-2013 DMD & Becker

Muscular Dystrophy


angiotensin   (1-7)[A(1-7)] 07-25-2013 DMD


deflazacort 08-16-2013 DMD


I – N-Gene Research Laboratories

N-Gene Research Laboratories, a privately held US biopharmaceutical company located in New York City, focuses on areas of metabolic disorders. The company receives a FDA ODD for DMD in January 2013.

II – Prosensa

The FDA grants ODD to Prosensa’s 4 preclinical compounds for DMD in January 2013 :

  1. PrO045
  2. PRO052
  3. PRO053
  4. PRO055.

PRO045 is currently in a Phase I/IIa dose-escalating safety study to assess its safety and efficacy. The compound induces exon 45 skipping in the dystrophin gene and could be suitable for an estimated 8% of all DMD patients. 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 and receives in June 2013, the FDA Breakthrough Therapy Designation in June 2013.

Other Prosensa activities in the news:

•   In August 2013, is awarded an EUR $6 million (approximately US $8.2 million) Framework Programme 7 (FP7) research grant from the European Union (EU) to support an ongoing clinical study of PRO045

•   In September 2013, announces the dosing of the 1st patient in a Phase I/II clinical trial of PRO053

•   In September 2013, announces primary endpoint in Phase III clinical trial of Drisapersen is not met

•   In November 2013, announces enrollment of 100th patient in Natural History Study of DMD

•   In November 2013, is awarded with Newcastle University (UK), an EUR $6 million (approximately US $8.2 million) Framework Programme 7 (FP7) research grant to support the development of imaging biomarkers for DMD.

III – Italfarmaco

Italfarmaco, an Italian pharmaceutical group, receives in April 2013, FDA ODD for Givinostat, a HDAC inhibitor, for DMD. Givinostat is a Histone Deacetylases (HDAC) inhibitor, which blocks enzymes called HDACs, which are involved in either turning genes ‘off’ or ‘on’ in cells. Givinostat, by blocking HDAC enzymes, is expected to turn ‘on’ the follistatin gene, resulting in increasing follistatin protein in muscle cells. Thus, an increase in muscle mass and prevention of muscle degeneration is expected.

A 2013 journal article, “Preclinical Studies in the mdx Mouse Model of Duchenne Muscular Dystrophy with Histone Deacetylase Inhibitor Givinostat”, is published in Molecular Medicine. Per the Abstract, the journal article explores:

“… the effectiveness of long-term treatment with different doses of the HDAC inhibitor Givinostat in mdx mice—the mouse model of Duchenne Muscular Dystrophy… the long-term (3.5 months) exposure of 1.5-month-old mdx mice to optimal concentrations of Givinostat promoted the formation of muscles with increased cross-sectional area and reduced fibrotic scars … leading to an overall improvement of endurance performance in treadmill tests and increased membrane stability … these findings provide the preclinical basis for an immediate translation of Givinostat into clinical studies with DMD patients”.

European Medicines Agency (EMA) in July 2012 grants Givinostat ODD.

IV – US Biotest

US Biotest, a private biological research company in San Luis Obispo, California, receives in July 2013, a FDA ODD for angiotensin (1-7)[A(1-7)] for the treatment of DMD.

V – Marathon Pharmaceuticals, LLC

Marathon Pharmaceuticals, an Illinois-based company that manufactures and commercializes pharmaceutical products for patients with rare diseases, receives a FDA ODD on August 16, 2013, for Deflazacort for DMD. Deflazacort is a glucorticoid used as an anti-inflammatory and immunosuppressant. The drug’s potency is around 70-90% that of prednisone. It is not available in the United States. It is sold in the UK by Shire under the trade name Calcort; and is available in Brazil, India, and other countries.

At Parent Project Muscular Dystrophy’s (PPMD) December 12th “Duchenne Policy Forum”, Dr. McDonald (UC Davis) mentions that “recent data shows Deflazacort more effective than Prednisone – boys walk longer”.

Please Note: “Technician Transfer Liquid To Test Tube” courtesy of US National Institute of Mental Health.

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

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