The EMA and FDA Grant Orphan Drug Designation to Pharnext's PXT-3003 for the Treatment of Charcot-Marie-Tooth Disease Type 1A

Quotes

Daniel Cohen, M.D., Ph.D., chairman and chief executive officer of Pharnext, said, "FDA and EMA orphan drug designations for PXT-3003 recognize the value this novel oral therapeutic could bring to CMT 1A patients. 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."

Catherine Scart-Gres, M.D., chief medical officer, said, "We are very pleased to receive orphan drug designation for PXT-3003. If approved, PXT-3003 could be a significant therapeutic advance for patients suffering from CMT 1A which impairs quality of life and can lead to severe disabilities. PXT-3003 has shown promise in a Phase 2 study and we are now actively preparing the next clinical trial." 

About Orphan Drug Designation

The EMA may grant orphan drug designation to provide regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU) and where no satisfactory treatment is available.

The FDA may grant orphan drug designation to facilitate drug development for drugs that target conditions affecting fewer than 200,000 patients in the U.S. while providing a significant therapeutic advantage over existing therapies.

About Pharnext

Pharnext is an advanced clinical stage biopharmaceutical company discovering and developing new therapeutics that target multiple key disease pathways for orphan and common neurological diseases including Charcot-Marie-Tooth disease, Alzheimer's disease, amyotrophic lateral sclerosis and Parkinson's disease. These new therapeutics consist of low dose combinations of repositioned drugs selected using Pharnext's proprietary network pharmacology approach. The company's first two lead programs are PXT-3003 for the treatment of Charcot Marie Tooth type 1A (Phase 2 completed) and PXT-864 for Alzheimer's disease (Phase 2 ongoing) and other neurologic indications. Pharnext is supported by BPI France (French Public Investment Bank) and AFM (French Muscular Dystrophy Association).

For further information, please visit www.pharnext.com

Corporate Contact
Catherine Scart-Gres, M.D.
Chief Medical Officer
[email protected]

Media Contacts
Tony Russo, Ph.D.
Matt Middleman, M.D.
Russo Partners
T: +1-212-845-4251
T: +1-212-845-4272
[email protected]
[email protected]

References

¹ Foley C, Schofield I, Eglon G, Bailey G, Chinnery PF, Horvath R. Charcot–Marie–Tooth disease in Northern England. J Neurol Neurosurg Psychiatry 2012; 83: 572-573

² Shy ME, Chen L, Swan ER, Taube R, Krajewski KM, Herrmann D, Lewis RA, McDermott MP. Neuropathy progression in Charcot-Marie-Tooth disease type 1A. Neurology. 2008; 70(5): 378-383

³ Van Paaseen et al. Review. PMP22 related neuropathies: Charcot-Marie-Tooth disease type 1A and Hereditary Neuropathy with liability to Pressure Palsies. Orphanet Journal of Rare Diseases9:38 (2014).