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Europe Grants Orphan Drug Status to JR-441, ERT for Sanfilippo A
The European Commission has granted an orphan drug designation to JR-441, JCR Pharmaceuticals’ brain-penetrating experimental enzyme replacement therapy (ERT) for Sanfilippo syndrome type A, the company has announced. The designation is given to investigative therapies with the potential to be safe and effective for rare, life-threatening, or chronically debilitating conditions that have no approved treatments, or where the potential therapy is showing significant benefit over existing treatments.
It is meant to accelerate JR-441’s clinical development and review by providing regulatory support and financial benefits. It also provides a 10-year marketing exclusivity period in Europe upon regulatory approval, if granted. JCR is planning to launch a global clinical trial testing the therapy in the first half of 2023. Sanfilippo syndrome type A, also known as mucopolysaccharidosis type IIIA, is caused by the absence or reduced activity of the heparan N-sulfatase enzyme due to mutations in the SGSH gene.
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