close

Products

Date: 2014-01-08

Type of information: Granting of the orphan status in the EU

Product name: GS010- adeno-associated viral vector containing the human NADH dehydrogenase 4 gene

Compound: adeno-associated viral vector containing the human NADH dehydrogenase 4 gene

Therapeutic area: Rare diseases - Genetic diseases - Ophtalmological diseases

Action mechanism: gene therapy

Company: Gensight-Biologics (France)

Disease: Leber's hereditary optic neuropathy (LHON)

Latest news:

  • • On 13 May 2011, orphan designation was granted by the European Commission to Institut de la Vision, France, for adeno-associated viral vector containing the human NADH dehydrogenase 4 gene for treatment of Leber\'s hereditary optic neuropathy. The sponsorship was transferred to Gensight-Biologics, France, in September 2013.

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: 2013-11-20

Orphan status UE: 2011-05-19 and 2013-10-03

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes