AGLE - Aeglea Bio's ACN00177 nabs Rare Pediatric status for inherited metabolic disorder

The FDA has designated has granted Rare Pediatric Disease tag to Aeglea BioTherapeutics' (AGLE) ACN00177 for the treatment of Homocystinuria, a metabolic disorder in which the body is unable to process certain building blocks of proteins (amino acids ) properly, and is characterized by elevated plasma homocysteine.Rare Pediatric Disease designation provides for the issuance of a priority review voucher following FDA approval. The voucher can be used for accelerated approval of a future application or it can be sold to a third party.Aeglea initiated a Phase 1/2 trial earlier this year to investigate ACN00177 for Homocystinuria.In October, the investigational therapy received Orphan drug tags in US and Europe.

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Aeglea Bio's ACN00177 nabs Rare Pediatric status for inherited metabolic disorder