Date: 2016-09-28
Type of information: Granting of the orphan status in the US
Product name: DTX401
Compound: recombinant adeno-associated virus serotype 8 vector encoding human glucose-6-phosphatase-alpha (G6Pase or G6PC)
Therapeutic area: Rare diseases - Genetic diseases
Action mechanism: gene therapy. DTX401 is an adeno-associated virus (AAV) gene therapy targeting the liver. DTX401 delivers stable expression and activity of the enzyme glucose-6-phosphatase (G6Pase). DTX401 has been shown to improve G6Pase activity and reduce hepatic glycogen levels, a well-described biomarker of disease progression, in multiple in vivo preclinical models.
Company: Dimension Therapeutics (USA - MA)
Disease: glycogen storage disease type Ia (von Gierke Disease)
Latest news: * On September 28, 2016, the FDA granted orphan drug designation for DTX401, a recombinant adeno-associated virus serotype 8 vector encoding human glucose-6-phosphatase-alpha (G6Pase or G6PC) for the treatment of glycogen storage disease type Ia (von Gierke Disease).
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: 2016-09-28
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
