EMA Agrees to Pediatric Investigation Plan to Assess PXT30003 as a Treatment for Children with CMT1A

The European Medicines Agency has agreed to Pharnext SA’s pediatric investigation plan for PXT3003, an investigational therapy being developed to treat Charcot-Marie-Tooth disease type 1A (CMT1A).

Because CMT1A typically appears in the first 20 years of a person’s life, Pharnext intends to explore the use of PXT3003 as an early intervention for children. The EMA requires a pediatric investigation plan to be submitted before the application for marketing authorization approval. This plan ensures that the necessary data is obtained in clinical studies to support the approval of a medication for children.

“The EMA agreement with our PIP represents a significant step forward in our efforts to bring PXT3003 to patients in Europe,” Daniel Cohen, MD, PhD, co-founder and CEO of Pharnext, said in a press release.

“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. In our Phase 2 study, PXT3003 was observed to stabilize and slow the progression of CMT1A, and also demonstrated clinical improvement in patients. 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,” he said.

Pharnext previously reported that PXT3003 stabilized and slowed the progression of CMT1A and demonstrated clinical improvement in patients in a Phase 2 trial (NCT01401257).

An update from an ongoing pivotal Phase 3 trial in adults with CMT1A (NCT02579759) was announced in late June, with top-line results expected in October 2018. Patients enrolled in the study are eligible to participate in an extension trial (NCT03023540) to evaluate the long-term safety and tolerability of PXT3003.

The Phase 3 trial protocols were amended during the course of the studies with no delays projected for their completion.

PXT3003 was developed using Pharnext’s proprietary technology called Pleotherapy, which uses artificial intelligence to identify combinations of existing medications, called Pleodrugs, which can act on several disease pathways simultaneously and offer increased safety and effectiveness to treat a particular disease.

PXT3003 has been granted orphan drug status in the U.S. and EU. Orphan drug status provides incentives to companies developing therapies for rare diseases that have an unmet need. The company also recently announced that PXT3003 was granted priority review by the China Food and Drug Administration.

CMT1A patients suffer from progressive muscle atrophy, or wasting away of muscles, in the legs and arms causing problems with walking, running, balance, and hand function.  The first symptoms usually appear during adolescence and evolve progressively throughout life.

There are an estimated 125,000 cases in the U.S. and the EU. To date, medications that alleviate symptoms or cure the disease have been approved and treatment consists of supportive care such as orthotics, leg braces, physical and occupational therapy or surgery.