Date: 2014-10-20
Type of information: Granting of the orphan status in the US
Product name: RST-001 - non-replicating recombinant adeno-associated virus vector containing a fragment of the gene encoding channel rhodopsin-2 protein
Compound: non-replicating recombinant adeno-associated virus vector containing a fragment of the gene encoding channel rhodopsin-2 protein
Therapeutic area: Ophtalmological diseases - Rare diseases
Action mechanism: gene therapy. RST-001is a first-in-class gene therapy application of optogenetics designed to restore vision to those affected by retinitis pigmentosa.
Company: RetroSense Therapeutics (USA - MI)
Disease: retinitis pigmentosa
Latest news: * On October 20, 2014, the FDA has granted orphan drug designation for non-replicating recombinant adeno-associated virus vector containing a fragment of the gene encoding channel rhodopsin-2 protein for the treatment of retinitis pigmentosa.
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: 2014-10-20
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
