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13.02.2014

Promising cream for butterfly children (EBS) receives "orphan designation" status

A clinical pilot study, undertaken at the EB House research unit in Salzburg, was able to show that the Diacerein cream resulted in better stability of the skin and reduced the formation of painful blisters in EBS-affected patients.

There is no cure at present for Epidermolysis bullosa (EB) but healing and alleviating approaches are being intensively researched world-wide. Current treatment is limited to the alleviating of symptoms, careful dressing of wounds and relieving pain. Yet here as well, new, even more effective options are needed.

The research team of the EB House Austria has now conducted laboratory tests on the active substance, Diacerein, a molecule of the rhubarb root, for its effect on EB simplex (EBS). The promising results were then confirmed in a clinical pilot study with EBS-affected patients and published. (Wally et al. Orphan J Rare Dis, 2013).

In early January, the efforts of Dr. Verena Wally and Prof. Johann Bauer bore fruit. The Diacerein cream which they had themselves developed was given the so-called "orphan designation" by the European Medicines Agency (EMA). This signifies a special status for the development of new medications and therapies for those affected by rare diseases.

The clinical pilot study was able to show that the Diacerein cream resulted in better stability of the skin and reduced the formation of painful blisters in EBS-affected patients. With the "orphan designation", the further development of this new medication can now be expedited in a large European study. The regulatory approval is expected in 2016 or thereabouts.

Background facts regarding the EMA and the orphan designation

The European Medicines Agency or EMA is a decentralised agency of the European Union in London. Since 1995, it has been responsible for the scientific evaluation of medicinal products. Its task is to ensure public health through the evaluation and monitoring of medicinal products in the EU.

The EMA has created the "orphan designation" for promising new treatment methods for patients affected by rare diseases. It is awarded only after very strict conditions are met and following a precise testing procedure. The goal is to at least partially compensate the commercial risks of the developer by way of subsidies and market exclusivity. Thus, the cost-intensive development of medication even for smaller groups of affected patients is of interest to the pharmacology industry. In this way, the chances that medicinal products for rare diseases become marketable are considerably higher.

Barbara Dissauer, DEBRA Austria

Yellow tube of Diacerein cream