Date: 2017-05-16
Type of information: Granting of the orphan status in the EU
Product name: SB-525
Compound: recombinant adeno-associated viral vector, AAV2 serotype 6 (rAAv2/6), expressing human factor 8 (hf8) cDNA
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Action mechanism:
- gene therapy/genome editing product. Sangamo has developed an adeno-associated virus (AAV) carrying a clotting Factor VIII gene construct driven by Sangamo's proprietary synthetic liver specific promoter, which in preclinical studies is more potent than existing AAV-based cDNA constructs currently under evaluation for the treatment of hemophilia A.
- SB-525 is being developed as part of an exclusive, global collaboration and license agreement with Pfizer.
Company: Sangamo Therapeutics (USA - CA) previously known as Sangamo Biosciences
Disease: hemophilia A
Latest news:
- • On May 23, 2017, the European Medicines Agency (EMA) has granted orphan medicinal product designation to SB-525, a clinical stage cDNA gene therapy candidate for hemophilia A.
- • On May 16, 2017, Sangamo Therapeutics announced that the FDA has granted Fast Track designation to SB-525, the company's clinical stage cDNA gene therapy candidate for hemophilia A. The FDA has cleared an Investigational New Drug application for this program, and a Phase 1/2 clinical trial evaluating SB-525 in adults with hemophilia A is expected to open and begin screening subjects for enrollment by the end of the second quarter 2017. Data from this study are expected in late 2017 or early 2018.
- • On May 3, 2017, the FDA has granted orphan drug designation for SB-525 (recombinant adeno-associated viral vector, AAV2 serotype 6 (rAAv2/6), expressing human factor 8 (hf8) cDNA) for treatment of hemophilia A. The FDA has cleared an IND for this program, and a Phase 1/2 clinical trial evaluating SB-525 in adults with Hemophilia A is expected to be opened and to begin screening subjects for enrollment later this quarter.
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: 2017-05-03
Orphan status UE: 2017-05-23
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
