US regulators have granted a Fast Track designation to a potential gene therapy treatment for haemophilia A from Pfizer and Sangamo.
SB-525, which also has orphan drug status from the FDA, is one of Sangamo’s four lead candidates and is expected to enter a Phase I/II clinical trial within the next six weeks.
Pfizer signed a global collaboration and licence deal with Sangamo for SB-525 and a number of its other gene therapy programmes for haemophilia just last week.
The pharma giant said then that SB-525 has best-in-class potential and would build on its own heritage in rare diseases and gene therapy.
SB-525 uses a recombinant adeno-associated virus (rAAV) to deliver a human Factor VIII cDNA construct and a proprietary promoter via a single.
The FDA awards Fast Track designations to treatments for serious conditions that could fulfil an unmet medical need.
Developers then benefit from a greater amount of interactions with the regulator, thus improving the chances of selected drugs going to receive Priority Review.