Date: 2015-08-10
Type of information: Granting of the orphan status in the EU
Product name: EGT-301 - adeno-associated viral vector serotype 9 containing the human iduronate-2-sulfatase gene
Compound: adeno-associated viral vector serotype 9 containing the human iduronate-2-sulfatase gene
Therapeutic area: Rare diseases - Genetic diseases
Action mechanism: gene therapy
Company: Laboratorios del Dr. Esteve (Spain)
Disease: mucopolysaccharidosis type II (Hunter's syndrome)
Latest news: * On July 16, 2015, the FDA has granted orphan drug designation for adeno-associated virus serotype 9 vector containing human Iduronate-2-sulfatase transgene for the treatment of mucopolysaccharidosis type II (Hunter syndrome). * On 14-16 July 2015, the Committee for Orphan Medicinal Products (COMP) adopted a positive opinion recommending adeno-associated viral vector serotype 9 containing the human iduronate-2-sulfatase gene for designation as orphan medicinal product for treatment of mucopolysaccharidosis type II (Hunter's 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: 2015-07-16
Orphan status UE: 2015-08-10
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
