Date: 2013-01-24
Type of information: Granting of the orphan status in the EU
Product name: EGT-201 - adeno-associated viral vector serotype 9 containing the human N-acetylglucosaminidase alpha gene
Compound: adeno-associated viral vector serotype 9 containing the human N-acetylglucosaminidase alpha gene
Therapeutic area: Rare diseases - Genetic diseases
Action mechanism: gene therapy
Company: Laboratorios del Dr. Esteve (Spain)
Disease: mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)
Latest news: * On December 6, 2012, the Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending adeno-associated viral vector serotype 9 containing the human N-acetylglucosaminidase alpha gene for designation as an orphan medicinal product for treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome).
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: 2012-12-27
Orphan status UE: 2013-01-24
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
