Date: 2017-01-25
Type of information: Granting of the orphan status in the EU
Product name: GS030 - adeno-associated viral vector serotype 2.7m8 containing the ChrimsonR-tdTomato gene
Compound: adeno-associated viral vector serotype 2.7m8 containing the ChrimsonR-tdTomato gene
Therapeutic area: Rare diseases - Genetic diseases - Ophtalmological diseases
Action mechanism:
- gene therapy. GS030 is based on optogenetics, a technology that makes cells responsive to light. GS030 consists of
a gene therapy product comprising a gene encoding a photoactivatable channelrhodopsin protein, delivered via a modified AAV2 (AAV2 7m8) and biomimetic goggles that stimulate the engineered retinal cells. The images are projected by a light source that uses a specific wavelength onto the retina. Once this optogenetic and photoactivable protein is expressed, it confers a photoreceptor-like function to the target cell, enabling the restoration of vision in patients with extremely reduced vision or who are blind due to retinitis pigmentosa.
Company: GenSight Biologics (France)
Disease: retinitis pigmentosa
Latest news:
- • On January 25, 2017, the FDA has granted orphan drug designation to Gensight's GS030 for the treatment of retinitis pigmentosa. GS030 is currently undergoing a Good Laboratory Practices regulatory toxicity study, and is expected to enter the clinic with a Phase I/II clinical trial in retinitis pigmentosa patients in Q3 2017, subject to toxicity results and future regulatory review.
- • On June 14-16, 2016, the Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending adeno-associated viral vector serotype 2.7m8 containing the ChrimsonR-tdTomato gene for designation as an orphan medicinal product 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: 2017-01-25
Orphan status UE: 2016-07-18
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
