Type of information: Granting of the orphan status in the US
Product name: LX2006
Compound: Adeno-associated viral vector encoding the human frataxin (FXN) gene
Therapeutic area: Rare diseases - Neurodegenerative diseases
Action mechanism: gene therapy. IV-administered, adeno-associated virus (AAV)-mediated gene therapy encoding the human frataxin gene.
Company: Lexeo Therapeutics (USA-NY)
Disease: Friedreich's Ataxia
Latest news: • On June 30, 2021, Lexeo Therapeutic announced that FDA has granted Rare Pediatric Disease designation and Orphan Drug designation to LX2006 for the treatment of Friedreich’s ataxia (FA). LX2006 cover cardiac disease and broader symptoms associated with FA.
Lexeo plans to initiate a Phase I/II clinical trial of LX2006 in patients with cardiomyopathy associated with FA in 2021.
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2021-06-24
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
Pediatric exclusivity UE: