Sangamo's Accelerated Fabry Gene Therapy Pathway Boosted by FDA Alignment

Sangamo Therapeutics has secured an agreement with the FDA for an accelerated approval pathway for its Fabry disease gene therapy, isaralgagene civaparvovec, promising a potential market entry reduction of three years. This alignment allows the use of existing Phase I/II study data, bypassing the need for a further registrational study, and leveraging the estimated glomerular filtration rate (eGFR) slope as an intermediate endpoint to seek approval[1][2]. This strategic move by Sangamo, supported by promising preliminary data, signifies a crucial step in delivering a much-needed treatment alternative for Fabry patients, enhancing its profile among potential partners and stakeholders[2].