Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a rare immune-mediated peripheral neuropathy characterized by progressive sensory loss, weakness, and reduced reflexes due to demyelination. It is treatable but can cause substantial long-term disability if diagnosis and treatment are delayed.
argenx reported $1.3B in Q1 2026 global product net sales (63% YoY growth). The FDA PDUFA target date for seronegative gMG is May 10, 2026. Positive ADAPT OCULUS results support an oMG label expansion. Pipeline milestones include myositis readout in Q3 2026 and MMN readout in Q4 2026.
Three FcRn inhibitors are approved for generalized myasthenia gravis, with a head-to-head trial of efgartigimod and nipocalimab underway. Emerging therapies including telitacicept, gefurulimab, and CAR-T cell therapy (KYV-101) show promise, while Immunovant's batoclimab failed TED trials.
Regulatory incentives for rare disease treatments, including market exclusivity and development subsidies, are spurring pharmaceutical innovation across the US, Europe, Japan, and Australia, with multiple companies advancing therapies for conditions affecting limited patient populations.
Dianthus Therapeutics will host a conference call on March 9, 2026, to discuss interim responder analysis results from Part A of the Phase 3 CAPTIVATE trial of claseprubart in Chronic Inflammatory Demyelinating Polyneuropathy (CIDP).
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07719153 |
Ultrasound-driven Stratification in CIDP |
NOT_YET_RECRUITING | |
| NCT07638566 |
A Study to Assess the Correct Dose, Safety and Efficacy of Empasiprubart in Adolescent Participants Aged 12 to Less Than 18 Years With Chronic Inflammatory Demyelinating Polyradiculoneuropathy |
NOT_YET_RECRUITING | PHASE2/PHASE3 |
| NCT07264426 |
Real-World Efgartigimod Effectiveness in CIDP: A Prospective Study |
RECRUITING | |
| NCT07091630 |
A Study to Assess the Efficacy and Safety of Empasiprubart in Adults With CIDP |
RECRUITING | PHASE3 |
| NCT07006597 |
Vitaccess Real CIDP Registry |
NOT_YET_RECRUITING | |
| NCT06920004 |
A Study to Assess Efficacy and Safety of Empasiprubart Versus IVIg in Adults With CIDP |
RECRUITING | PHASE3 |
| NCT06714838 |
Rituximab Induced Remission in Patients With Chronic Inflammatory Demyelinating Polyneuropathy |
RECRUITING | PHASE3 |
| NCT05723848 |
Electronic Monitoring of Disease Activity in Patients With Chronic Inflammatory Demyelinating Polyneuropathy |
COMPLETED | |
| NCT05614128 |
Validation of Cutaneous Nerve Demyelination in Diagnosis and Treatment of CIDP |
TERMINATED | |
| NCT05584631 |
IVIG vs SCIG in CIDP |
RECRUITING | PHASE1 |
