Date: 2018-08-27
Type of information: Granting of the orphan status in the US
Product name: AAV-CNGA3 - adenovirus associated viral vector serotype 2/8 containing the human CNGA3 gene
Compound: adenovirus associated viral vector serotype 8 containing the human Cyclic Nucleotide Gated Channel Alpha 3 subunit (CNGA3) gene
Therapeutic area: Rare diseases - Genetic diseases - Ophtalmological diseases
Action mechanism:
- gene therapy. ACHM is an inherited retinal disease that severely limits a person’s sight by preventing cone photoreceptors in the eye from functioning. Individuals with ACHM are often legally blind from birth, have extreme sensitivity to light, and experience involuntary eye movements. AAV-CNGA3 is an investigational gene therapy treatment designed to restore cone function, delivered to the cone receptors at the back of the eye via subretinal injection.
Company: MeiraGTx (USA - NY)
Disease: achromatopsia caused by mutations in the CNGA3 gene
Latest news:
- • On August 27, 2018, MeiraGTx announced that the Offices of Orphan Products Development and Pediatric Therapeutics of the FDA have granted rare pediatric disease designation to its gene therapy product candidate AAV-CNGA3 for the treatment of patients with achromatopsia (ACHM) due to mutations in the CNGA3 gene.
- • On August 7, 2018, the FDA has granted orphan drug designation for its AAV-CNGA3 gene therapy product candidate for the treatment of achromatopsia (ACHM) caused by mutations in the CNGA3 gene.
- • On July 31, 2018, the European Commission has granted orphan drug designation of AAV-CNGA3 for the treatment of achromatopsia (ACHM) caused by mutations in the CNGA3 gene
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: 2018-08-07
Orphan status UE: 2018-07-31
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
