Date: 2016-10-14
Type of information: Granting of the orphan status in the EU
Product name: SGT-001 - adeno-associated viral vector differentially expressing microdystrophin 5 gene via the muscle-specific promoter CK8
Compound: adeno-associated viral vector differentially expressing microdystrophin 5 gene via the muscle-specific promoter CK8
Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases
Action mechanism: gene therapy
Company: Solid GT (USA - MA)
Disease: Duchenne muscular dystrophy
Latest news: * On October 24, 2016, Solid Biosciences and its subsidiary, Solid GT, announced that the FDA and the European Commission have granted Orphan Drug designations for SGT-001, for the treatment of patients with Duchenne muscular dystrophy (DMD). Solid plans to initiate clinical studies for SGT-001 in 2017. * On 6-8 September, 2016, the Committee for Orphan Medicinal Products (COMP) has recommended the granting of an orphan designation for recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter for treatment of Duchenne muscular dystrophy. * On August 31, 2016, the FDA has granted orphan drug designation for SGT-001 (adeno-associated viral vector differentially expressing microdystrophin 5 gene via the muscle-specific promoter CK8) for the treatment of Duchenne muscular dystrophy.
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: 2016-08-31
Orphan status UE: 2016-10-14
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
