RNA Therapy for Rare Skin Disease Gets Orphan Status

dystrophic epidermolysis bullosa proqr rna therapy

The EMA has granted orphan drug designation to ProQR’s QR-313, a possible first disease-modifying treatment for the rare disease dystrophic epidermolysis bullosa.

Dystrophic epidermolysis bullosa (DEB) is a genetic disease that causes the skin to easily blister, severely affecting the quality of life of patients since a very early age. Current treatment is limited to dressing the blisters and taking antibiotics to avoid infections.

The Dutch biotech ProQR wants to put an end to it and develop a treatment that directly addresses the genetic cause of the disease. Its drug candidate QR-313 is an RNA therapy aimed at providing a functional copy of the COL7A1 gene that is mutated in patients with epidermolysis bullosa. In particular, the treatment is aimed at patients with a mutation in exon 73 of the gene, the most common cause of the disease.

The EMA’s orphan drug designation for QR-313 comes just two months after the FDA’s. The treatment will enter clinical trials in 2018, and thanks to its orphan status, it will be able to fare faster through the regulatory process in both Europe and the US in order to help those with epidermolysis bullosa, who currently have no effective treatment options.

Image via Anton Nalivayko /Shutterstock


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