Products

Date: 2018-01-19

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

Product name: SB-913

Compound: adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and the human iduronate 2-sulfatase (hIDS) gene

Therapeutic area: Rare diseases - Genetic diseases

Action mechanism: gene therapy/genome editing product

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

Disease: mucopolysaccharidosis type II (MPS II)

Latest news:

• • On December 7, 2017, the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA) has issued a positive opinion on the application for orphan medicinal product designation (OMPD) for SB-913 (adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human iduronate 2-sulfatase gene), Sangamo's genome editing product candidate for the treatment of mucopolysaccharidosis Type II (MPS II).
• • On March 1, 2017, the FDA has granted orphan drug designation for SB-913 (adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and the human iduronate 2-sulfatase (hIDS) gene) for treatment of mucopolysaccharidosis type II (MPS II), a rare lysosomal storage disorder. Sangamo has also submitted an application to the FDA for rare pediatric disease designation for SB-913.
• In 2017, Sangamo is conducting a Phase 1/2 clinical trial evaluating SB-913 as an in vivo genome editing treatment for MPS II.

 

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: 2017-02-27

Orphan status UE: 2018-01-19

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes