PRIME status for ProQR's sepofarsen
New York-traded shares in ProQR Therapeutics (Nasdaq: PRQR) looked set to open around 6% up on Monday with news that the European Medicines Agency (EMA) has granted PRIority MEdicines (PRIME) status to a drug in development for Leber’s congenital amaurosis 10 (LCA10).
ProQR is developing RNA medicines for the treatment of severe genetic diseases, with sepofarsen being developed to target the p.Cys998X mutation in the CEP290 gene in LCA10, the most common cause of blindness due to genetic disease in children.
The Dutch biotech firm sees the European Union as an important market for this medicine, and the company can take confidence from the fact that an RNA-interfering treatment has already been approved in Europe, in Alnylam Pharmaceuticals’ (Nasdaq: ALNY) Onpattro (patisiran).
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