The FDA Grants Fast Track Designation to
Pharnext’s PXT3003 for CMT1A
Pharnext, an advanced clinical-stage biopharmaceutical company, created a drug combination of three drugs: baclofen, naltrexone and sorbitol known as PXT3003. Baclofen is known to relieve muscle spasms by relaxing the muscles.  Naltrexone is used to help assist in the treatment of opioid addiction.  Sorbitol is a sugar alcohol.   Pharnext set out to prove that the combination of these drugs reduces the PMP22 levels in people with CMT1A.

The data hasn’t been released yet to show the affect on PMP22 in humans, but the data does show that patients generally felt better and functioned better.  The method which they used to test patients’ function pre- and post-trial focuses on the gross motor skills like thigh mobility and shoulder mobility (can you brush your hair, kick a ball, etc.) and didn’t as specifically measure fine motor skills like picking up a penny or buttoning buttons on a shirt.

So while patients felt statistically better, the big question is, “does this cocktail reduce the PMP22 levels in humans?”

You might ask, “Who cares?  If I feel better, it could be a good option”  And you’d be right.

PXT3003 could be the first drug marketed exclusively to CMT1A patients which can demonstrate improvement in patients’ symptoms.  The FDA’s fast track designation accelerates the development and review of therapies with the potential to treat serious conditions for which there is no cure. The designation allows more frequent communication with the FDA to discuss the therapy’s development and review process.

We are excited because this proves that CMT is a focus of pharma companies and hundreds of top-notch researchers.  This drug also brings CMT to the attention of the FDA.  However, the data released is not yet convincing that this is the cure we are hoping for; we will wait for the release of more detailed data to tell us that.  Still, this is a very promising development from Pharnext and we are grateful for their attention to CMT.