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