In the fight against common diseases such as cancer the strategy is clear: scientists are working flat out on research, methods of early detection and treatments. That the demand is enormous is not surprising: according to the Federal Statistics Office, around 26 percent of all deaths in Germany in 2011 were due to cancer. So it is no wonder that patients, researchers and pharmaceutical companies want to see effective drugs developed fast.
But what is the situation with drugs to treat diseases which affect only a few people? “These drugs are often neglected by pharmaceutical companies because they are economically unattractive,” says Dr. Karin Aufenvenne, a biologist, who personally experienced the problem over the past few years. Around one year ago she and Prof. Heiko Traupe, senior physician for dermatology at the skin clinic at Münster University Hospital, achieved a breakthrough in treating a special form of ichthyosis with medication. This highly unpleasant skin disease, often known as fish-scale disease, is caused by genetic defects and occurs directly after birth. Patients suffer from an extreme thickening of the top layer of the skin, which manifests itself in a dark brown, lamellar desquamation covering the entire body. “Around 2,500 people in Europe are estimated to have the disease – which means it is ultra-rare,” says Karin Aufenvenne.For this reason no pharmaceutical company could be found which was prepared to progress and - above all - market the therapy developed at Münster University (WWU) in close cooperation with the working group headed by Dr. Margitta Dathe at the Leibniz Institute of Molecular Pharmacology (FMP) in Berlin. This situation could, however, change shortly. The reason is that the medication was recently awarded orphan drug status by the European Union – an unprecedented event at WWU and FMP. With the official EU designation as a “drug to treat rare afflictions”, it becomes more economically attractive. “Pharmaceutical companies that buy the rights to such a drug do not have to pay any approval costs, for example,” explains Karin Aufenvenne. “And after regulatory approval they have exclusive rights to the drug for ten years.” In Münster there is a strong desire to find a company attracted by these incentives. “The distress felt by patients who have this disease is enormous – not only due to the physical symptoms, but also because of the mental suffering.”
Dr. Christina Heimken | idw
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