BioDiem Ltd
Melbourne, Thursday 9 September 2010: Australian biopharmaceutical development company BioDiem Ltd (ASX: BDM) today announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug designation to BDM-E for the treatment of Retinitis Pigmentosa.
BioDiem plans to take advantage of the incentives offered under the Orphan Drug program. The Company will be looking to undertake an early stage clinical trial to establish proof-of-concept in Retinitis Pigmentosa and is seeking partners and funding assistance to this end.
To achieve orphan drug designation for BDM-E, BioDiem submitted an application to the FDA outlining its scientific documentation to demonstrate the rarity and severity of the medical condition and the potential benefit of BDM-E in treating this condition.
BioDiem’s preclinical work has demonstrated BDM-E’s potential for the treatment of Retinitis Pigmentosa. In vivo studies have shown that BDM-E is able to reduce the degree of retinal damage of the retinal cell layers. It has also been shown to exert a degree of protection of the retina in models of this disease. In another research model of light damage it prevented photoreceptor apoptosis (programmed cell death) when high doses were administered. Through the use of BDM-E in Russia and results of an earlier clinical trial BDM-E has already shown a good safety profile.
Retinitis Pigmentosa is a serious eye disease which causes progressive degeneration of the delicate light receptor cells in the retina, which, over time, diminishes night and peripheral vision and eventually leads to blindness. This condition affects between 1 in 3,500 to 4,000 people, equivalent to 77,000 and 88,000 people in the USA. Currently, there is no therapy that stops the evolution of the disease or restores the vision.
The FDA Orphan Drug program is intended to support the clinical development of new drugs in rare diseases affecting less than 200,000 people in the United States. A drug designated as an “orphan” must still undergo the full regulatory review process to achieve marketing authorization, however there are opportunities for applicants to seek assistance in this process via the Office of Orphan Product Development (OOPD). Other major incentives include the possibility of study design assistance, exemption from application filing fees, grant funding for clinical trials, tax credits for clinical research and seven years of US market exclusivity after marketing approval.
BioDiem’s CEO, Julie Phillips, said: “We are delighted to receive FDA Orphan Drug Designation for our BDM-E product. Our strategy is to co-develop or outlicence this product initially for the US market and then consider expansion into other indications.”
-ENDS-
About BioDiem Ltd
BioDiem is an ASX-listed company, based in Melbourne, with an international focus on finding, adding value to and commercialising world-class research for vaccines, infectious diseases and other therapeutic areas. The company uses a cost-efficient approach to drug development through collaborations with academic centres of excellence, contract research organizations and partnerships with international pharmaceutical companies.
BioDiem’s leading product is the Live Attenuated Influenza Vaccine (LAIV) technology, a novel intranasal vaccine being developed to prevent infection from endemic and pandemic influenza. The technology was licensed to BioDiem by the Institute of Experimental Medicine in St Petersburg. In 2004, BioDiem licensed the LAIV technology to Nobilon International B.V. (now part of Merck & Co, Inc.). It is currently in Proof of Concept (Phase II) stage clinical trials as part of its development for European registration. It has also been launched in India as NasoVac for protection against H1N1 influenza.
About BDM-E and Retinitis Pigmentosa
BDM-E is a tetrapeptide currently in clinical use in Russia on a compassionate use basis for treating a range of eye diseases including Retinitis Pigmentosa and dry age-related macular degeneration. BioDiem has licensed BDM-E outside Russia and has performed in vitro and animal studies of BDM-E at the Universities of Cambridge, Wisconsin, Melbourne and Monash as a prelude to clinical studies. In vitro studies reveal complex effects of BDM-E on cell proliferation, apoptosis and inflammatory mediators. The studies suggest that BDM-E may increase repair of retinal tissues, and animal models of clinical retinal diseases support this hypothesis.
Retinitis Pigmentosa is a type of progressive retinal dystrophy, a group of inherited disorders in which abnormalities of the photoreceptors (rods and cones) or the retinal pigment epithelium of the retina lead to progressive visual loss. Affected individuals first experience defective dark adaptation or nyctalopia (night blindness), followed by reduction of the peripheral visual field (known as tunnel vision) and, sometimes, loss of central vision late in the course of the disease. This disorder is characterized by the progressive loss of photoreceptor cells and may eventually lead to blindness. Retinitis Pigmentosa is one of the most common forms of inherited retinal degeneration.
For additional information, please visit www.biodiem.com