FDA Grants Fast Track Designation to Pharnext’s PXT3003 for CMT1A

Pharnext‘s PXT3003 was granted fast track designation for Charcot-Marie-Tooth disease type 1A (CMT1A) by the U.S. Food and Drug Administration, the company announced.

“We are pleased to receive Fast Track designation for PXT3003 in CMT1A” Daniel Cohen, MD, PhD, co-founder and CEO of Pharnext, said in a press release.

“CMT1A is a rare, chronic neuropathy that affects at least 125,000 people across the U.S. and Europe. There is currently no approved treatment for this disease. We look forward to continuing our productive dialogue with the FDA to rapidly progress towards a U.S. New Drug Application for PXT3003, to deliver this therapy to patients as quickly as possible.”

PXT3003 is an investigational combination of baclofen, naltrexone, and sorbitol — three already approved treatments that act on the nervous system — formulated as an oral solution that is given twice a day. The therapy was also granted orphan drug designation in the European Union and the U.S., and priority review in China.

In preclinical studies, PXT3003 inhibited the overexpression of the PMP22 gene, whose duplication causes CMT1A, and protected the nerve cells, relieving neuromuscular symptoms. A Phase 2 clinical trial conducted with 80 CMT1A patients suggested that the treatment was safe and tolerable.

In December 2015, Pharnext launched a pivotal, randomized, double-blind Phase 3 trial called PLEO-CMT (NCT02579759) to assess the safety and efficacy of PXT3003 in 323 CMT1A patients ages 16 to 65 years. The treatment was well-tolerated and eased disability in patients with mild to moderate CMT1A.

The open-label, follow-up extension study PLEO-CMT-FU (NCT03023540) started in March 2017 to asses the long-term safety and efficacy of the compound in patients who had completed PLEO-CMT. The study is currently ongoing.

The FDA’s fast track designation is geared toward accelerating 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.

Pharnext is a biopharmaceutical company that focuses on the development of therapies for neurodegenerative diseases. It uses an innovative approach called Pleotherapy to find combinations of previously approved medications that in low, optimized doses can treat conditions that lack specific therapies.