The U.S. Food and Drug Administration has granted Fast Track designation to SAR446268, Sanofi's (SNY,SNYNF,SAN.PA) investigational one-time AAV gene therapy targeting non-congenital forms of myotonic dystrophy type 1 (DM1), including juvenile and adult-onset cases. This designation is intended to accelerate the development and review of therapies that address serious medical conditions and meet unmet treatment needs.
Myotonic dystrophy type 1 (DM1) is a rare, genetic disorder that causes progressive muscle weakness and wasting, with no currently approved medicines.
SAR446268 is currently under investigation in a first-in-human, phase 1-2 study to evaluate the safety, tolerability, and efficacy (clinical study identifier: NCT06844214). The first patient is planned for enrolment in late 2025. Sanofi has already been granted orphan designations for SAR446268 in both the US (July 2024) and European Union.
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