Katairo GmbH from Kusterdingen near Tübingen is developing a new treatment for a rare form of macular degeneration. The active substance “Remofuscin” is able to remove the toxic lipofuscin from cells, thus enabling Stargardt’s Disease to be treated successfully for the first time. The company in the STERN BioRegion has been awarded orphan medicinal product designation for Remofuscin. Orphan drug designation was awarded in November 2013 following after comprehensive testing by the European Medicines Agency (EMA), thus underpinning the therapeutic potential of the medical substance.

“Katairo is delighted to receive the orphan drug designation. It is an important milestone on the way to obtaining financing,” says Dr. Wolfgang Klein, CEO of Katairo GmbH. “In addi-tion to the benefits this brings for development and marketing, it is excellent to have our approach endorsed by the regulatory authorities.” Being awarded orphan drug designation by the EMA means that the company not only receives free protocol assistance for further development, but is also granted exclusive sales rights for the substance in Europe.

The term “orphan” refers to rare medical conditions for which the pharmaceutical industry undertakes virtually no development of medicinal products, as the cost of bringing them to market and obtaining approval would usually not be recovered by the expected sales of the products. To ensure that patients suffering from rare conditions receive the best possible treatment, companies dedicated to researching and developing medical products for these conditions receive special support.

Stargardt’s Disease is one of these rare conditions. It is a form of macular degen-eration that can also lead to blindness in young people. As with the dry form of age-related macular degeneration (AMD), this condition causes toxic lipofuscin consisting of metabolite waste that cannot be excreted by the cells to accumulate in the cell layer of the retinal pigment epithelium. This causes photoreceptors in the retina to die. There is currently no treatment capable of halting the death of photoreceptors or even stimulating their regeneration. Low vision aids are currently the only solution for easing the situation in the short term.

Katairo GmbH was founded by Prof. Ulrich Schraermeyer and other partners in 2011. Prof. Schraermeyer is an expert in the pathology of the retinal pigment epithelium, the layer of cells in the eye that is affected by pathological changes. Last year, his range of treatments involving “Remofuscin” for the dry form of age-related macular degeneration (AMD) was awarded the 2012 Innovation Award of the biotechnology regions in Germany.