PXT3003: Lessons for therapeutic development in Charcot-Marie-Tooth disease type 1A and related neuropathies.

In a September 2026 review, Joshua Burns traces the history of PXT3003, a combination of baclofen, naltrexone and sorbitol for CMT1A, prompted by a Phase 3 trial in China published in the same journal. He describes the drug’s formulation problems, interrupted development, regulatory uncertainty and disputed efficacy, and argues that judging it depends on how much of the evidence is public.

This is a review, not new trial data.