BRIEF — EMA agrees to Pharnext's PIP for PXT3003

Pharnext, a biopharma with a new approach to developing drug combinations based on big genomic data and artificial intelligence, has 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, chief executive 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."

PXT3003 is being evaluated in an international pivotal Phase III 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 USA.