Last reviewed · How we verify
PXT3003 dose 1
PXT3003 is a fixed-dose combination of three active pharmaceutical ingredients that work synergistically to reduce neuroinflammation and promote nerve regeneration in peripheral neuropathies.
PXT3003 is a fixed-dose combination of three active pharmaceutical ingredients that work synergistically to reduce neuroinflammation and promote nerve regeneration in peripheral neuropathies. Used for Charcot-Marie-Tooth disease type 1A (CMT1A).
At a glance
| Generic name | PXT3003 dose 1 |
|---|---|
| Also known as | DOSE 1, DOSE 2, (RS)-baclofen, naltrexone hydrochloride and D-sorbitol oral fixed dose combination, liquid oral solution |
| Sponsor | Pharnext S.C.A. |
| Drug class | Fixed-dose combination / Immunomodulator |
| Target | Multiple targets (baclofen: GABA-B receptor; naltrexone: opioid receptors; sorbitol: osmotic agent) |
| Modality | Small molecule |
| Therapeutic area | Neurology / Rare Genetic Disorders |
| Phase | Phase 3 |
Mechanism of action
PXT3003 combines baclofen, naltrexone, and sorbitol in a proprietary formulation designed to modulate immune responses and reduce inflammatory markers in the peripheral nervous system. The combination targets multiple pathways involved in demyelinating neuropathies, particularly Charcot-Marie-Tooth disease type 1A (CMT1A), by reducing neuroinflammation and potentially promoting myelin repair and axonal regeneration.
Approved indications
- Charcot-Marie-Tooth disease type 1A (CMT1A)
Common side effects
- Dizziness
- Nausea
- Fatigue
- Headache
Key clinical trials
- Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PHASE3)
- Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A (PHASE3)
- Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT) (PHASE3)
- Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- PXT3003 dose 1 CI brief — competitive landscape report
- PXT3003 dose 1 updates RSS · CI watch RSS
- Pharnext S.C.A. portfolio CI