Date: 2016-12-12
Type of information: Granting of the orphan status in the EU
Product name: adeno-associated viral vector serotype 8 encoding engineered rhodopsin DNA-binding repressor and human rhodopsin expression cassettes
Compound: adeno-associated viral vector serotype 8 encoding engineered rhodopsin DNA-binding repressor and human rhodopsin expression cassettes
Therapeutic area: Rare diseases - Genetic diseases - Ophtalmological diseases
Action mechanism: gene therapy
Company: FondazioneTelethon (Italy)
Disease: retinitis pigmentosa
Latest news: * On 3-4 November 2016, the Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending the approval of the orphan medicinal product designation for adeno-associated viral vector serotype 8 encoding engineered rhodopsin DNA-binding repressor and human rhodopsin expression cassettes for treatment of retinitis pigmentosa.
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:
Orphan status UE: 2016-12-12
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
