Pfizer ceases development of Hemophilia A gene therapy Giroctocogene Fitelparvovec
Pfizer
Richmond: Sangamo Therapeutics, Inc., a genomic medicine company, has announced it will regain development and commercialization rights to giroctocogene fitelparvovec, an investigational gene therapy product candidate for the treatment of adults with moderately severe to severe hemophilia A that it has co-developed with, and licensed to Pfizer Inc., following a decision by Pfizer to terminate the global collaboration and license agreement between the parties.
Sangamo intends to explore all options to advance the program, including seeking a potential new collaboration partner. Pfizer has indicated to Sangamo that this termination reflects its decision not to proceed with the Biologics License Application (BLA) and Marketing Authorisation Application (MAA) submissions for, or to pursue commercialization of, giroctocogene fitelparvovec.
In July 2024, Pfizer announced positive topline results from the Phase 3 AFFINE trial evaluating giroctocogene fitelparvovec that demonstrated the trial met primary and key secondary objectives of superiority compared to prophylaxis. OnDecember 9, 2024, Pfizer presented detailed AFFINE data in an oral presentation at the 66th ASH Annual Meeting and Exposition, with the presentation selected as part of the “Best of ASH” program, which highlights the most cutting-edge science presented during the Annual Meeting. In November 2024 Pfizer indicated to Sangamo that Pfizer was discussing these data with regulatory authorities. Pfizer previously indicated to Sangamo that anticipated BLA and MAA submissions were expected to occur in early 2025.
“Giroctocogene fitelparvovec has demonstrated the potential to be a life changing gene therapy treatment for hemophilia A patients, and following positive results from the Phase 3 AFFINE trial, we believe it is well positioned for regulatory submissions and potential commercialization,” said Sandy Macrae, Chief Executive Officer of Sangamo Therapeutics. “While we were surprised and extremely disappointed by Pfizer’s decision to end our collaboration so close to the anticipated BLA and MAA submissions, especially given the compelling pivotal clinical trial data, we appreciate their collaboration in leading a robust and successful clinical development program and for advancing the asset to this important stage. We are committed to exploring the optimal path forward for this important treatment, including seeking the right partner with the focus and understanding of the genomic medicine commercial environment to bring this medicine to patients. In parallel, we remain focused on advancing our wholly owned neurology genomic medicine pipeline and progressing our Fabry gene therapy program towards a potential BLA submission in the second half of 2025.”
The collaboration and license agreement with Pfizer will terminate effective April 21, 2025, at which time Pfizer will be required to transition the giroctocogene fitelparvovec program back to Sangamo. All trial participants will continue to be monitored as planned during the transition period.
"Sangamo believes that its recently announced partnerships with Genentech and Astellas, and advanced business development discussions for its Fabry gene therapy program, will allow it to chart a path forward for its neurology genomic medicine pipeline as it prepares to initiate expected enrollment of patients in the Phase 1/2 study of ST-503 for idiopathic small fiber neuropathy in mid-2025, and file an anticipated Clinical Trial Authorisation (CTA) submission for the prion disease program in Q4 2025, each subject to securing adequate additional funding," the release stated.
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