Pharnext’s PXT3003 Granted Priority Review in China for Charcot-Marie-Tooth Type 1A

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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The French biopharmaceutical company Pharnext recently announced that its candidate for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A) was granted priority review by the China Food and Drug Administration (CFDA).

PXT3003 is a fixed-dose combination of baclofen, naltrexone, and sorbitol. The therapy candidate has received orphan drug status in both the U.S and Europe.

“We are pleased that PXT3003 has been identified as a priority by the CFDA, and that its fast-track status will decrease the waiting time for the drug’s approval,” Daniel Cohen, MD, PhD, co-founder and chief executive officer of Pharnext, said in a press release.

Pharnext says it is developing a new approach to therapy combinations based on big genomic data and artificial intelligence. Known as Pleotherapy, the approach seeks synergic combinations of treatments, called Pleodrugs, that offer several advantages including increased effectiveness and safety, the company said.

The first-in-class Pleodrug PXT3003 is currently being tested in the Phase 3 PLEO-CMT trial (NCT02579759) in Europe and the United States, with results anticipated by the end of the year.

The PLEO-CMT trial is studying the safety and effectiveness of two doses of PXT3003 compared to placebo in 323 patients with mild to moderate CMT1A.

In March 2017, researchers initiated a follow-up study, called PLEO-CMT-FU (NCT03023540) in which all patients who were treated for the first 15 months in the first trial are receiving PXT3003 treatment for an additional nine months to assess the long-term safety and tolerability of the drug.

Updates presented at the 2017 Peripheral Nerve Society Meeting in Barcelona, Spain, showed that the therapy candidate had been well-tolerated and effective in improving patients’ disability in previous Phase 2 studies in adults with CMT1A.

The priority review in China will significantly shorten the wait for clinical approval and expedite registration as an imported pharmaceutical if data confirm the treatment is beneficial to patients with CMT1A.

The commercialization of PXT3003 for CMT1A in greater China is owned by GeneNet, a joint venture between Pharnext and Talsy. The greater China region comprises mainland China, Hong Kong, Taiwan, and Macau. GeneNet also retains exclusive licensing rights to all PXT3003 patents applied for and authorized in this world region.