December 2011- GoFAR is glad to announce that in December, Repligen received regulatory approval from the Italian Istituto Superiore della Sanità and the Ethic Committee of the San Luigi Hospital (Orbassano, Torino) where the study will run, to initiate a Phase 1 safety study in patients next year. While this approval is an important first step, there are a number of activities which need to be completed before the clinical site will be able to enroll patients. The study will evaluate the pharmacokinetic and safety profile of escalating doses RG2833 in up to 20 FRDA patients and changes in frataxin levels which may enable Repligen to gain early insight into the potential benefit of treating patients with RG2833. This will be the first clinical trial of a novel drug specifically designed to treat FRDA.
Repligen previously received U.S Fast Track and European Orphan Medicinal Product designations for RG2833 for Friedreich’s Ataxia.
Additional information is anticipated to be available on Repligen website when the study is ready to enroll patients.