FDAnews Drug Daily Bulletin
Pharmaceuticals / Submissions and Approvals

Pharnext’s Charcot-Marie-Tooth Disease Treatment Gets Fast Tracked

Feb. 11, 2019
A A

The FDA granted a fast track designation to Pharnext’s Pleodrug PXT3003, a treatment for Charcot-Marie-Tooth disease.

The French drugmaker’s twice-daily, novel synergistic drug — a combination of naltrexone, sorbitol and baclofen — is in development to treat Type 1A of the disease, a rare chronic neuropathy that currently has no approved treatment. The condition affects at least 125,000 individuals in the U.S. and Europe.

The company’s Phase 3 PLEO-CMT trial, a 15-month double blind study, met the FDA and EMA’s primary endpoint of overall neuropathy limitation scale and exhibited a statistically significant difference when compared to placebo.

View today's stories