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DRUGSFORD receives Orphan Drug Designation for their LP-DF003 compound

23. April 2015

Rare diseases of the retina, such as Retinitis pigmentosa (RP), cause the photoreceptors of the eye to die. The consequences are severe vision loss or even blindness, not the least amongst young people, and unfortunately there is still no treatment for these diseases. The so called Orphan Drug Designation (ODD) relates to special legislation in Europe for rare and severe diseases, which facilitates clinical testing and provides for 10 years of exclusive market access.

DRUGSFORD, a Collaborative Project funded by the European Commission under the 7th Framework Programme (HEALTH-F2-2012-304963), aims to develop a drug and drug delivery system combination for the treatment of RP, up to the point where the combination is ready to enter clinical testing. The DRUGSFORD consortium was formed in late 2012 by three academic institutions, the University of Tubingen, the University of Modena and Reggio Emilia, and the University of Lund, as well as two small-to-medium enterprise partners, with expertise in cyclic guanosine monophosphate (cGMP) analogue synthesis (Biolog Life Science Institute, Bremen) and drug delivery systems (to-BBB, Leiden), respectively.
The DRUGSFORD concept relies on the fact that RP often involves dysregulated cGMP signalling in dying photoreceptors. This can be counteracted by synthetic cGMP analogues, which are delivered to the photoreceptors across the blood-brain-barrier.

Through testing in a variety of RP models, DRUGSFORD has identified a candidate cGMP analogue and delivery system combination, named LP-DF003. Importantly, treatment with LP-DF003 resulted in structural and functional rescue of diseased retinas in RP animal models. These very promising effects have now resulted in the European Medicines Agency (EMA) granting an Orphan Drug Designation to LP-DF003 (EU/3/15/1462; ec.europa.eu/health/documents/community-register/html/o1462.htm).

In obtaining an ODD for its first lead compound formulation, the DRUGSFORD consortium has made a significant step towards the clinical development of a new treatment for RP and has strongly improved the prospects for future commercialisation of LP-DF003.

[Further information may be obtained from the DRUGSFORD consortium´s website: www.drugsford.eu]