Posted by Michael Wonder on 05 Feb 2019
Pharnext announces FDA fast track designation for Pleodrug PXT3003 for the treatment of Charcot-Marie-Tooth disease type 1A
4 February 2019 - Pharnext announced today that the U.S. FDA has granted fast track designation for the development of PXT3003 for the treatment of patients with Charcot-Marie-Tooth disease type 1A.
PXT3003 is a novel synergistic combination of baclofen, naltrexone and sorbitol, formulated as an oral solution that is given twice a day.
In December 2015, Pharnext initiated the PLEO-CMT study, a pivotal 15-month, double-blind Phase 3 study that assessed the efficacy and safety of PXT3003 in 323 patients with Charcot-Marie-Tooth disease type 1A aged 16 to 65 years.
