Date: 2017-09-27
Type of information: Granting of the orphan status in the US
Product name: AMT-130
Compound: adeno-associated viral vector serotype 5 encoding a microRNA targeted to human huntingtin gene
Therapeutic area: Rare diseases - Genetic diseases - Neurodegenerative diseases
Action mechanism:
Company: Uniqure (The Netherlands)
Disease: Huntington's disease
Latest news:
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2017-09-27
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news: