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Date: 2017-05-03

Type of information: Granting of the orphan status in the US

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 8 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.

Company: Sangamo Therapeutics (USA - CA) previously known as Sangamo Biosciences

Disease: hemophilia A

Latest news:

  • • 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:

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes