Sangamo secures FDA’s Fast Track Designation for haemophilia candidate
pharmafile | May 17, 2017 | News story | Research and Development, Sales and Marketing | FDA, Pfizer, Sangamo Therapeutics
Sangamo Therapeutics has announced that the FDA has granted its cDNA gene therapy candidate for haemophilia A Fast Track Designation. The candidate, SB-525, is set to enter clinical trials, at Phase 1/2 level, this year with the results expected by late 2017, early 2018.
SB-525 uses a recombinant adeno-associated virus to deliver a human Factor VIII cDNA construct and proprietary, synthetic liver-specific promoter to the nucleus of liver cells with a single infusion. The therapy is designed as a single treatment strategy intended to provide continuous, therapeutic expression of Factor VIII protein.
Early studies, in non-human primates, have investors excited that this particular treatment could become best-in-class for haemophilia A.
The news comes only a week after Sangamo and Pfizer had entered into a potential $545 million deal. Pfizer was attracted to the licensing partnership as a result of Sangamo’s four lead products, one of which includes SB-525. The deal involved $70 million in cash up-front, with a further $475 million possible in milestone payments.
The deal also meant that should SB-525 prove successful in clinical trials, Pfizer will take over the work of developing the candidate beyond Phase 1/2, as well as being responsible for manufacturing and commercialisation activities.
“With a long-standing heritage in rare disease, including hemophilia, Pfizer is an ideal partner for our Hemophilia A program,” said Dr. Sandy Macrae, Sangamo’s Chief Executive Officer, on the announcement of the partnership. “We believe Pfizer’s end-to-end gene therapy capabilities will enable comprehensive development and commercialization of SB-525, which could potentially benefit Hemophilia A patients around the world. This collaboration also marks an important milestone for Sangamo as we continue to make progress in the translation of our ground-breaking research into new genomic therapies to treat serious, genetically tractable diseases.”
Ben Hargreaves
Related Content
FDA approves ImmunityBio’s Anktiva bladder cancer treatment
ImmunityBio has announced that the US Food and Drug Administration (FDA) has approved Anktiva (N-803, …
EC approves Pfizer’s Emblaveo for multidrug-resistant infection treatment
Pfizer has announced that the European Commission (EC) has granted marketing authorisation for Emblaveo (aztreonam-avibactam) …
Roche’s Alecensa approved by FDA as lung cancer treatment
Roche has announced that the US Food and Drug Administration (FDA) has approved Alecensa (alectinib) …
