Sanofi's SAR446268 Receives FDA Fast Track for DM1 Treatment

The US FDA has granted fast track designation to Sanofi’s SAR446268, an experimental gene therapy for myotonic dystrophy type 1 (DM1).

This rare progressive neuromuscular disease affects about 1 in 2300 people globally, and has no approved treatments available right now.

The FDA fast track process is designed to expedite the development and review of therapies for serious diseases and unmet medical needs, giving hope to DM1 patients.

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SAR446268 is targeting the source of DM1 by using a vectorized RNA interference (RNAi) method to silence the DMPK gene with a single administration. The therapy aims to silence toxic DMPK RNA transcripts in DM1 by removing abnormal DMPK RNA foci which disrupt normal muscle splicing. This mechanism could restore muscle function and improve key DM1 symptoms of progressive muscle weakness, myotonia, and other multi-system complications affecting the heart, lungs and endocrine systems.

SAR446268 is now being developed for a first-in-human phase 1-2 clinical trial (NCT06844214) to assess the safety, tolerability, and efficacy, with first patient enrollment expected by late 2025. The therapy has also received orphan drug designation in the U.S. and EU. This recognition showcases the potential to change outcomes and care for this rare disease.

Sanofi, an R&D-driven biopharma utilizing AI technologies, is relentless in the quest to avoid diabetes for patients with rare diseases.  The fast track designation from the FDA highlights the need and opportunity of SAR446268 as a potential gamechanger for DM1 patients.