PXT3003 Shows Sustained Safety, Benefits for Patients With CMT1A

High-dose PXT3003, an investigational therapy for Charcot-Marie-Tooth disease type 1A (CMT1A), continues to show long-term safety and efficacy, according to an early analysis of a Phase 3 extension study. All participants showed improvements on the Overall Neuropathy Limitation Scale (ONLS), a measure of physical disability. “These new results…

Pharnext Shares Top-Line Results from Pivotal Phase 3 Studies of PXT3003; More to Come

Pharnext recently provided an update from its ongoing Phase 3 clinical trials evaluating PXT3003 for treating Charcot-Marie-Tooth disease type 1A (CMT1A) in adults, and also announced that some top-line results are expected by October 2018. PXT3003 is a fixed-dose combination of baclofen, naltrexone and sorbitol with orphan…

Pharnext’s PXT3003 Granted Priority Review in China for Charcot-Marie-Tooth Type 1A

The French biopharmaceutical company Pharnext recently announced that its candidate for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A) was granted priority review by the China Food and Drug Administration (CFDA). PXT3003 is a fixed-dose combination of baclofen, naltrexone, and sorbitol. The therapy candidate has received orphan drug status in both…