Sophia Antipolis, France
Nicox S.A. (Euronext Paris: COX) today announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for naproxcinod for the treatment of Duchenne Muscular Dystrophy (DMD). ODD is a status granted to drugs or biological products that treat rare diseases or conditions. The designation qualifies the sponsor of the drug for various development incentives, including a period of US marketing exclusivity upon marketing approval for the designated indication, potential tax credits and the waiver of certain fees1.
Naproxcinod is a CINOD (Cyclooxygenase-Inhibiting Nitric Oxide-Donating) anti-inflammatory candidate currently under evaluation by an undisclosed financial partner for potential clinical development in DMD2. Nicox has granted the undisclosed partner the exclusive right, should the results of the evaluation be satisfactory to the partner, to invest at the end of the evaluation period in naproxcinod and next generation nitric oxide (NO)-donors outside ophthalmology through an independent structure.
DMD is the most common and serious form of muscular dystrophy, a group of inherited diseases that cause muscle weakness and muscle loss. Naproxcinod already showed promising preclinical results in models of muscular dystrophy3 and received a European Orphan Drug Designation for the treatment of DMD in October 2013.
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References
1. For more information, please visit the FDA website: http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/default.htm.
2. See Nicox press release dated February 14, 2014.
3. Long-term treatment with naproxcinod significantly improves skeletal and cardiac disease phenotype in the mdx mouse model of dystrophy, Uaesoontrachoon K, Quinn JL, Tatem KS, Van der Meulen JH, Yu Q, Phadke A, Miller BK, Gordish-Dressman H, Ongini E, Miglietta D, Nagaraju K. Hum Mol Genet. 2014, 15; 23(12):3239-49.