Oxford BioMedica’s gene therapy for Usher syndrome wins orphan drug status from EU agency

Oxford BioMedica’s (LSE: OXB) UshStat gene therapy for Usher syndrome 1B has received orphan drug designation from the European Medicines Agency (EMEA). European ‘orphan drugs’ benefit from incentives, including ten years of marketing exclusivity and reduced regulatory fees. Clinical development of UshStat is expected to start in 2011, in collaboration with Sanofi-Aventis (NYSE: SNY).

Usher syndrome 1B is an inherited condition that results in hearing loss and progressive loss of vision from retinitis pigmentosa. UshStat is designed to deliver a corrected version of abnormal Myosin VIIA (MYO7A) gene, which causes the defect, into the cells of the retina using the company's LentiVector gene delivery technology.

“This designation is an important step towards the start of clinical trials of UshStat for Usher syndrome 1B as part of our landmark collaboration with Sanofi-Aventis to develop gene therapies in the field of ophthalmology", Oxford BioMedica CEO John Dawson said, "We are delighted to have received orphan designation from the European Medicines Agency for another of our innovative LentiVector-based candidates targeting debilitating and progressive ocular diseases."

The European Medicines Agency grants orphan drug designation to products that potentially provide significant advantages over current treatments for life-threatening or chronically debilitating conditions, affecting up to five in 10,000 people in the European Union.

Oxford BioMedica has a total of four Sanofi-Aventis collaborations, using the LentiVector gene delivery technology in its pipeline: the StarGen, RetinoStat, UshStat and EncorStat therapies. The StarGen therapy for Stargardt Disease received orphan drug designation from the EMEA in December 2009. StarGen and RetinoStat are expected to start clinical development in 2010.  http://www.proactiveinvestors.co.uk/companies/news/12415/oxford-biomedicas-gene-therapy-for-usher-syndrome-wins-orphan-drug-status-from-eu-agency-12415.html