Drug Discovery and Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Women in Pharma and Biotech
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • Resources
    • Video features
    • Podcast
    • Voices
    • Webinars
  • Pharma 50
    • 2025 Pharma 50
    • 2024 Pharma 50
    • 2023 Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50
  • Advertise
  • SUBSCRIBE

EMA Agrees With Pharnext’s Pediatric Investigation Plan For PXT3003

By Pharnext SA | July 11, 2018

Pharnext SA, a biopharmaceutical company developing drug combinations based on big genomic data and artificial intelligence, announced that the European Medicines Agency (EMA) has agreed with its pediatric investigation plan (PIP) for PXT3003 in Charcot-Marie-Tooth disease type 1A (CMT1A).

“The EMA agreement with our PIP represents a significant step forward in our efforts to bring PXT3003 to patients in Europe,” said Daniel Cohen, M.D., Ph.D., co-founder and chief executive officer of Pharnext. “Given that much of the progression of CMT1A occurs in the first two decades of patients’ lives, we believe that by intervening in childhood, we can have a greater impact on patients’ disease trajectory.

See related article on PLEOTHERAPY, including PXT3003: “Two Drugs Can Be Better Than One,” an in-depth report featuring Rodolphe Hajj, Ph.D., chief pharmacology officer at Pharnext.

“In our Phase 2 study, PXT3003 was observed to stabilize and slow the progression of CMT1A, and also demonstrated clinical improvement in patients, ” Cohen added. “We are dedicated to providing the estimated 14,000 children in Europe living with CMT1A a safe therapeutic option that may offer significant long-term relief.”

As part of the regulatory process for registering new medicines with the EMA, pharmaceutical companies are required to provide a PIP that outlines the clinical development strategy for studying the investigational product in children. EMA agreement with the PIP is required before a company can file a marketing authorization application for any new medicinal product in Europe.

PXT3003 is being evaluated in an international pivotal Phase 3 clinical trial in adults with CMT1A, with top-line results expected by October 2018. The clinical study investigating the safety and efficacy of PXT3003 for CMT1A in children will be conducted in Europe, Canada, and the United States.

(Source: Pharnext SA; Pictured: Pharnext headquarters in the southwestern suburban area of Paris, France, courtesy Pharnext)


Filed Under: Drug Discovery

 

Related Articles Read More >

EVEREST lead investigator on why Dupixent sets a new bar for treating coexisting CRSwNP and asthma
Sanders, King target DTC pharma ads but the industry worries more about threats to its $2B R&D model
Zoliflodacin wins FDA nod for treatment of gonorrhea
FDA approved ENFLONSIA for the prevention of RSV in Infants
“ddd
EXPAND YOUR KNOWLEDGE AND STAY CONNECTED
Get the latest news and trends happening now in the drug discovery and development industry.

MEDTECH 100 INDEX

Medtech 100 logo
Market Summary > Current Price
The MedTech 100 is a financial index calculated using the BIG100 companies covered in Medical Design and Outsourcing.
Drug Discovery and Development
  • MassDevice
  • DeviceTalks
  • Medtech100 Index
  • Medical Design Sourcing
  • Medical Design & Outsourcing
  • Medical Tubing + Extrusion
  • Subscribe to our E-Newsletter
  • Contact Us
  • About Us
  • R&D World
  • Drug Delivery Business News
  • Pharmaceutical Processing World

Copyright © 2025 WTWH Media LLC. All Rights Reserved. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media
Privacy Policy | Advertising | About Us

Search Drug Discovery & Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Women in Pharma and Biotech
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • Resources
    • Video features
    • Podcast
    • Voices
    • Webinars
  • Pharma 50
    • 2025 Pharma 50
    • 2024 Pharma 50
    • 2023 Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50
  • Advertise
  • SUBSCRIBE