Date: 2018-01-19
Type of information: Granting of the orphan status in the EU
Product name: SB-318
Compound: adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and the human alpha-L-iduronidase (hIDUA) gene
Therapeutic area: Rare diseases - Genetic diseases
Action mechanism:
- gene therapy. Using Sangamo's zinc finger nuclease (ZFN) genome editing technology, SB-318 is designed as a single treatment strategy intended to provide stable, continuous production of the alpha-L-iduronidase (IDUA) enzyme for the lifetime of the patient.
Company: Sangamo Therapeutics (USA - CA) previously known as Sangamo Biosciences
Disease: mucopolysaccharidosis type I (MPS I)
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 for SB-318 (adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human alpha liduronidase gene) for the treatment of mucopolysaccharidosis type I (MPS I).
- • On February 27, 2017, Sangamo Therapeutics announced that the FDA has granted rare pediatric disease designation for SB-318, an in vivo genome editing product candidate for the treatment of mucopolysaccharidosis type I (MPS I), a lysosomal storage disorder.
- In 2017, Sangamo is conducting a Phase 1/2 clinical trial evaluating SB-318 for MPS I. The FDA previously granted SB-318 with orphan drug designation.
- Rare pediatric disease designation is granted to drugs and biologics intended to treat orphan diseases affecting less than 200,000 patients in the U.S., primarily age 18 years or younger. The designation provides incentives to advance the development of rare disease drugs, including access to the FDA's expedited review and approval programs. In addition, under the FDA's Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives approval for a new drug application or biologics license application for a rare pediatric disease may be eligible to receive a voucher for a priority review of a subsequent marketing application for a different product. The priority review voucher may be used by the sponsor or sold or transferred.
- • On January 9, 2017, the FDA has granted orphan drug designation for SB-318 (adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and the human alpha-L-iduronidase (hIDUA) gene) for treatment of mucopolysaccharidosis type I (MPS I).
- In 2017, Sangamo plans to conduct the first ever in vivo genome editing clinical trials including Phase 1/2 studies for SB-318 for the treatment of MPS I
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: 2017-01-09
Orphan status UE: 2018-01-19
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
