Pharnext’s treatment for Charcot Marie Tooth 1A

Published on March 19th, 2020


It is with great joy that we welcome Pharnext’s press release announcing that PXT3003 for the treatment of CMT1A has been granted Promising Innovative Medicine (PIM) by the UK health authorities

It means that PXT3003 is a promising candidate for the provision before obtaining marketing authorization accordingly to the Early Access to Medicines Scheme (EAMS).

Daniel Cohen, M.D., Ph.D., co-founder and Chief Executive Officer of Pharnext, said: “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.

It is important to remember that in 2014, the EMA and FDA granted orphan drug designation to PXT3003 for the treatment of CMT1A in adults. We are confident in the positive development of PXT3003 testing and marketing authorization.

For more information, read the press release:

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