Oxford Biomedica Regulatory News (OXB)

15 Dec 2009 07:00



OXFORD BIOMEDICA'S STARGEN™ FOR STARGARDT DISEASE RECEIVES EUROPEAN ORPHAN DRUG DESIGNATION

Oxford, UK - 15 December 2009: Oxford BioMedica (LSE: OXB), a leading gene therapy company, announced today that StarGen, the Company's gene-based therapy for Stargardt disease, has received orphan designation from the Committee for Orphan Medicinal Products of the European Medicines Agency (EMEA). Stargardt disease is a hereditary disorder of the eye that is caused by abnormalities in a gene called ABCA4 in the retina. StarGen is designed to deliver a corrected version of the ABCA4 gene into the cells of the retina using the Company's LentiVector® technology. In collaboration with sanofi-aventis, clinical development is expected to start in 2010. The US charity, Foundation Fighting Blindness, is also supporting the programme and previously funded the preclinical development.

The EMEA grants orphan drug designation to products that may provide a significant advantage over current treatments, if any exist, for life-threatening or chronically debilitating conditions affecting up to five in 10,000 people in the European Union. Companies with European orphan drug designation benefit from incentives, including ten years of marketing exclusivity and reduced regulatory fees.

Oxford BioMedica's Chief Executive Officer, John Dawson, commented: "There is a real and urgent need for an effective treatment of Stargardt disease. Gene correction offers the only means of addressing the root cause of this debilitating, sight-robbing disorder. The EMEA's decision to grant orphan designation to StarGen adds significant value by providing development, regulatory and commercial advantages. StarGen is one of the four gene-based therapies for ocular diseases that we are developing in collaboration with sanofi-aventis and it also benefits from the Foundation Fighting Blindness' valuable assistance. Both StarGen and our other lead ocular programme, RetinoStat® for wet age-related macular degeneration, are on track to enter clinical development in 2010." 

-Ends-

Notes to editors

1. Oxford BioMedica

Oxford BioMedica (LSE: OXB) is a biopharmaceutical company developing innovative gene-based medicines and therapeutic vaccines that aim to improve the lives of patients with high unmet medical needs. The Company's technology platform includes a highly efficient gene delivery system (LentiVector®), which has specific advantages for targeting diseases of the central nervous system and the eye; and a unique tumour antigen (5T4), which is an ideal target for anti-cancer therapy. Through in-house and collaborative research, Oxford BioMedica has a broad pipeline and its partners include sanofi-aventis, Sigma-Aldrich and Pfizer (formerly Wyeth). Technology licensees include Biogen Idec, GlaxoSmithKline, Merck & Co and Pfizer. Further information is available at www.oxfordbiomedica.co.uk

2. Stargardt disease

Stargardt disease is the most common genetic form of juvenile macular degeneration with an incidence of approximately 1.1 in 10,000, which is equivalent to an estimated 55,000 people in the European Union. Stargardt disease is caused by mutations in the ABCA4 gene, which produce a dysfunctional protein that cannot perform energy transport to and from photoreceptor cells in the retina. The photoreceptor cells then degenerate, leading to vision loss. Age of onset is typically 8-12 years with severely decreased visual acuities developing within nine years. Currently, there is no effective treatment for Stargardt disease. 

3. StarGen™

StarGen is a development-stage gene therapy for the treatment of Stargardt disease. The product is designed to replace the defective ABCA4 gene that causes Stargardt disease by using Oxford BioMedica's LentiVector® technology to deliver a corrected version of the gene into the cells of the retina. The generation of a functional protein from the corrected gene has the potential to reduce or even halt the ongoing retinal degeneration. A single administration of StarGen could provide long-term or potentially permanent stabilisation of ocular function. StarGen has demonstrated efficacy in the only available preclinical model of Stargardt disease. In this preclinical setting, a single administration of StarGen showed sustained efficacy for the duration of the six-month study.