Good News for CMT1A Patients – PXT3003

by | Jun 9, 2020 | 0 comments


pharnextI’m excited by Pharnext’s recent announcement that three major regulatory agencies in the United Kingdom, Europe and US have recognized PXT3003 as a lead drug candidate to treat CMT1A. 

The United Kingdom has granted Pharnext a Promising Innovative Medicine (PIM) designation as an early indication that a medicinal product is a promising candidate for the Early Access to Medicines Scheme (EAMS) in the treatment, diagnosis or prevention of life-threatening or seriously debilitating conditions with unmet need.

Despite the climate of COVID-19, our industry partner, Pharnext SA, continues to plan for the additional pivotal Phase 3 PXT3003 trial for CMT1A in patients 16 years and older in the US and Europe.

“All existing data indicate that PXT3003 is a safe and well tolerated drug combination. We look forward to continuing our discussions with U.S. and European regulatory authorities to advance the clinical development of PXT3003 and initiate as quickly as possible an additional pivotal Phase 3 trial in the U.S. and Europe.” 

–  Daniel Cohen, M.D., Ph.D., Co-Founder and Chairman of the Scientific Advisory Board of Pharnext

You may be eligible to participate. By creating a profile in HNF’s Global Registry in Inherited Neuropathies (GRIN), you may be considered as a potential candidate for the Phase 3 study. 


You can also help advance the knowledge of CMT and the impact on Quality of Life, by joining the CMT&Me App. The objective of the app is to better understand the impact of the disease on patients’ daily lives: the burden of CMT, its natural history and treatment, and medical, social and pharmacoeconomic effects.

Read full press release from Pharnext

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