Type of information: Granting of the orphan status in the EU
Product name: CB 2679d/ISU304
Compound: recombinant adeno-associated viral vector serotype 6 encoding the B-domain-deleted
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
- coagulation factor/protein. CB 2679d/ISU304 is a next-generation coagulation Factor IX variant. It has exhibited enhanced procoagulant activity, improved efficacy in inhibiting blood loss, and prolonged duration of action in bleeding and non-bleeding preclinical models compared with other Factor IX products on the market.
- Catalyst has a collaboration with ISU Abxis to advance the development of CB 2679d/ISU304 through a Phase 1/2 proof-of-concept study in individuals with severe hemophilia B. After Phase 1/2, ISU Abxis has an option for exclusive commercial rights in South Korea and a profit share on ex-South Korean commercialization. Catalyst retains full development and commercial rights for CB 2679d/ISU304 outside of South Korea.
Company: Catalyst Biosciences (USA - CA)/Voisin Consulting (France)
Disease: hemophilia B
- • On June 28, 2017, Catalyst Biosciences announced that the European Commission has granted orphan medicinal product designation to CB 2679d/ISU304, a clinical stage drug candidate for hemophilia B. A Phase 1/2 proof-of-concept study is currently ongoing in individuals with severe hemophilia B. Data from the trial are expected by the end of 2017.
- • On May 31, 2017, Catalyst Biosciences announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending orphan medicinal product designation for CB 2679d/ISU304 for the treatment of hemophilia B.
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA:
Orphan status UE: 2017-06-20
Pediatric exclusivit _USA:
Pediatric exclusivity UE: