Sanofi's SAR446268 Therapy Advances in the United States

Sanofi announced that its gene therapy SAR446268 has been granted fast-track approval by the U.S. FDA. This decision concerns the treatment of myotonic dystrophy type 1 (DM1), a rare genetic disorder that causes muscle weakness and wasting. Currently, no medication is approved for this condition.

SAR446268 uses vector-based RNA interference to target and silence DMPK gene expression. This novel approach could improve muscle function by restoring normal tissue splicing.

The treatment is in phase 1-2 clinical trials to assess its safety and efficacy. Sanofi, which has already been granted orphan status in the United States and the EU, aims to address an unmet medical need.