Date: 2015-11-18
Type of information: Granting of the orphan status in the US
Product name: Adenovirus-associated viral vector serotype 10 carrying the human N-sulfoglucosamine sulfohydrolase cDNA (SGSH)
Compound: Adenovirus-associated viral vector serotype 10 carrying the human N-sulfoglucosamine sulfohydrolase cDNA
Therapeutic area: Rare diseases - Genetic diseases
Action mechanism: gene therapy
Company: Lysogene (France)
Disease: mucopolysaccharidosis type IIIA (Sanfilippo A syndrome)
Latest news: * On November 18, 2015, the FDA has granted orphan drug designation for adeno-associated viral vector serotype 10 carrying the human N-sulfoglucosamine sulfohydrolase cDNA for treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome). * On November 11-13, 2014, the Committee for Orphan Medicinal Products (COMP) has recommended the granting of an orphan designation for adeno-associated viral vector serotype 10 carrying the human N-sulfoglucosamine sulfohydrolase cDNA for treatment of mucopolysaccharidosis type IIIA (Sanfilippo A 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: 2015-11-18
Orphan status UE: 2014-12-16
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
