Date: 2017-11-27
Type of information: Granting of the orphan status in the EU
Product name: BB-301 - genetically modified adeno-associated viral vector serotype 9 expressing shRNA as well as a codon-optimised shRNA-insensitive wildtype PABPN1
Compound: genetically modified adeno-associated viral vector serotype 9 expressing shRNA as well as a codon-optimised shRNA-insensitive wildtype PABPN1
Therapeutic area: Rare diseases - Genetic diseases
Action mechanism:
- gene therapy. BB-301 is a single vector system which uses DNA directed RNA interference (ddRNAi) to silence expression of the mutant gene associated with oculopharyngeal muscular dystrophy, while simultaneously adding back a copy of the normal version of the same gene to restore gene function.
Company: Clinipace (Germany) Benitec BioPharma (Australia)
Disease: oculopharyngeal muscular dystrophy
Latest news:
- • On November 27, 2017, Benitec Biopharma announced it has submitted an application with the FDA seeking orphan drug designation for BB-301 as a treatment of oculopharyngeal muscular dystrophy (OPMD).
- • On January 12, 2017, orphan designation was granted by the European Commission for genetically modified adeno-associated viral vector serotype 9 expressing shRNA as well as a codon-optimised shRNA-insensitive wildtype PABPN1 (also known as BB- 301) for the treatment of oculopharyngeal muscular dystrophy (EU/3/16/1816) .
- • On 6-8 December, 2016, the Committee for Orphan Medicinal Products (COMP) has recommended the granting of an orphan designation for genetically modified adeno-associated viral vector serotype 9 expressing shRNA as well as a codon-optimised shRNA-insensitive wildtype PABPN1 for treatment of oculopharyngeal 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:
Orphan status UE: 2017-01-12
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
