Date: 0000-00-00
Type of information: Granting of the orphan status in the US
Product name: SHP654 (BAX 888)
Compound:
Therapeutic area:
Action mechanism:
- • gene therapy. SHP654 (BAX 888) is an investigational factor VIII (FVIII) gene therapy intended to treat hemophilia A using a recombinant adeno-associated virus serotype 8 (rAAV8) vector to deliver a codon-optimized, B-domain deleted FVIII (BDD-FVIII). This gene therapy selectively targets the liver. It involves the delivery of a functional copy of FVIII to the body’s liver to enable its own production of FVIII, rather than relying on a factor-based treatment. The FVIII expression is further controlled in patients by incorporating the liver-specific transthyretin (TTR) promoter/enhancer.
- This gene therapy includes technology acquired from Chatham Therapeutics, a spin-out of Asklepios Biopharmaceutical.
Company:
Disease:
Latest news:
- • On September 18, 2017, the FDA awarded Orphan Drug Designation to Shire’s gene therapy candidate SHP654 (BAX 888), an investigational factor VIII (FVIII) gene therapy for the treatment of hemophilia A. The regulatory agency also granted Shire investigational new drug (IND) status for SHP654.
- Shire also received FDA clearance for the IND application it submitted earlier this year to initiate a global multi-center study with SHP654 to evaluate the safety and optimal dose needed to boost factor VIII activity levels and affect hemophilic bleeding. The company anticipates that the SHP654 Phase 1/2 study will begin by the end of 2017.
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: 2017-09-18
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
