The FDA and European Medicines Agency have given Pharnext SAS an orphan drug designation to its PXT-3003 for the treatment of Charcot-Marie-Tooth disease type 1A. PXT-3003 is an oral fixed low-dose combination of baclofen, naltrexone and sorbital.
The orphan drug designation allows PXT-3003 marketing exclusively for 7 years in the United States and 10 years in Europe if approved for Charcot-Marie-Tooth disease type 1A (CMT 1A). Other benefits include fee reductions, assistance in study design from the FDA and European Medicines Agency (EMA), potential for expedited drug development and eligibility for drug grants.
“FDA and EMA orphan drug designations for PXT-3003 recognize the value this novel oral therapeutic could bring to CMT 1A patients,” Daniel Cohen, MD, PhD, chairman and chief executive officer of Pharnext, stated in a company press release. “This is an important milestone that strengthens our ultimate commitment to develop and bring rapidly to market effective novel therapies based on low-dose combinations of repositioned drugs. This also provides additional confidence in our other programs currently in development.”