The company Pharnext SA, which is developing the investigational drug PXT3003 for the treatment of Charcot-Marie-Tooth disease type 1A, has agreed upon a paediatric investigation plan for the drug with the European Medicines Agency (EMA). The source article can be read here, at Business Wire.
About Charcot-Marie-Tooth Disease
Charcot-Marie-Tooth disease (CMT) is a term used to describe a group of conditions that are characterised by damage in the peripheral nerves. The condition is sometimes also called hereditary motor and sensory neuropathy. According to the NHS website, peripheral nerves are involved in processes such as muscle control and conveying sensory information such as touch. Damage to these nerves may cause weakness in certain areas (such as feet, legs, ankles, hands), numbness, and an unusual foot shape and/or gait.
Charcot-Marie-Tooth disease type 1A (CMT1A) is a form of the condition that, according to the NIH, is caused by having an extra copy of the PMP22 gene. Similarly to some other forms of CMT, people affected by CMT1A may experience muscle weakness and wasting, and sensory loss.
About the Paediatric Investigation Plan
A paediatric investigation plan (PIP) is part of the process of registering drugs with the EMA. The PIP should discuss the clinical development strategy that will be used when giving children the experimental drug. The EMA has to approve the PIP before any drug can be submitted for review using a marketing authorisation application.
Paediatric investigation plans typically include information about how the drug will be administered (e.g. as a liquid, pill, etc.) and what the needs of different age groups are, and other information.
PXT3003 is an experimental drug. According to a spokesperson for Pharnext, Phase 2 trials have found that it can slow the progression of CMT1A.
A further Phase 3 pivotal study is planned to take place in adults with CMT1A. The topline results of this study are expected to be made available around October 2018. A study that investigates the use of PXT3003 in children is planned to take place in Europe, the US, and Canada. Click here to see the original article for more information.