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Eidos Therapeutics receives orphan drug designation for AG10 from EMA.

Eidos Therapeutics announced that the Committee for Orphan Medicinal Products, or COMP, within the European Medicines Agency, or EMA, adopted a positive opinion for the designation of Eidos' product candidate, AG10, as an orphan medicinal product for the treatment of transthyretin, or TTR, amyloidosis, or ATTR. In addition, the EMA also granted a product-specific pediatric investigational plan waiver to Eidos for AG10. TTR normally circulates as a four-part molecule or tetramer, but in ATTR the molecule dissociates into individual monomers, which are unstable and aggregate as amyloid fibrils. Eidos' lead product candidate, AG10, is designed to target ATTR at its source by stabilizing tetrameric TTR in the blood.

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Publication:The Fly
Date:Oct 26, 2018
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