- The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to Avrobio's ( NASDAQ: AVRO ) gene therapy AVR-RD-02 to treat Gaucher disease (GD).
- Gaucher disease is a rare lysosomal disorder caused by buildup of certain fatty substances in organs, mainly in the spleen and liver.
- Avrobio's noted that AVR-RD-02 has previously received fast track status in the U.S., orphan drug designations in the U.S. and EU, and Innovative Licensing and Access Pathway designation in the U.K.
- AVRO +5.56% to $0.72 premarket Oct. 27
For further details see:
Avrobio stock rises on FDA rare pediatric disease tag for Gaucher disease gene therapy