Date: 2017-12-07
Type of information: Granting of the orphan status in the US
Product name: AAV2/5-OPTIRPE65 (A001) - adenovirus associated viral vector serotype 5 containing the human RPE65 gene
Compound: adenovirus associated viral vector serotype 5 containing the human RPE65 gene
Therapeutic area: Rare diseases - Genetic diseases - Ophtalmological diseases
Action mechanism:
- gene therapy. A001 is an adeno-associated virus (AAV2/5) investigational gene therapy designed to deliver a codon-optimized RPE65 cDNA under the control of a synthetic RPE-specific promoter to the back of the eye. A001 is delivered via subretinal injection of up to 1 ml to cover the largest possible area of viable retina.
Company: MeiraGTx (USA - NY)
Disease: Leber congenital amaurosis
Latest news:
- • On December 7, 2017, MeiraGTx announced that the Offices of Orphan Products Development and Pediatric Therapeutics of the FDA have granted rare pediatric disease designation to the Company’s gene therapy product candidate AAV2/5-OPTIRPE65 (A001) for the treatment of patients with Leber’s congenital amaurosis due to mutations in the RPE65 gene (LCA2). A001 previously received orphan drug designation from the FDA for the treatment of LCA2 in 2016.
- MeiraGTx is conducting an open label, multi-center Phase I/II dose escalation trial of A001 in up to 27 patients, aged 3 years and older, diagnosed with severe early onset LCA2. The primary endpoint of the study is to determine the safety of the treatment. Secondary endpoints include improvement in visual function including a mobility maze, retinal function, retinal structure and quality of life measures. MeiraGTx has completed the three dose escalation cohorts of adult patients in this study and in November 2017 the first pediatric patient was treated at the Moorfields Eye Hospital in London.
- In October 2017, the FDA accepted the Investigational New Drug (IND) application for A001 opening the path for the treatment of pediatric patients in the U.S.
- • On March 10, 2016, the FDA has granted orphan drug designation for adenovirus associated viral vector serotype 5 containing the human RPE65 gene for treatment of Leber congenital amaurosis.
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: 2016-03-10
Orphan status UE: 2015-11-11
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
