Mavorixafor
Orphan DrugFDA Approved
Description
Mavorixafor is an oral CXCR4 antagonist approved to increase neutrophil counts in certain patients with WHIM syndrome. It is a rare immunology drug with a highly specific inherited-disease market and a small eligible population. The product is notable for translating a long clinical-development program into a first-in-class orphan therapy.
Indications & Therapeutic Use
WHIM syndrome
Linked Diseases:
WHIM syndrome
C536697
Global Availability (1 countries)
| Country | Access Route | Regulatory Pathway | Lead Time | Status |
|---|---|---|---|---|
| — | — | — | — |
Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Mavorixafor
| Generic Name | mavorixafor |
| Brands | 1 brand available |
| Active Ingredient | mavorixafor |
| Drug Class | WHIM syndrome |
| Manufacturer | X4 Pharmaceuticals |
| Dosage Forms | Oral capsule |
| Medical Code | L04AX |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Not Required |
| Reg. Status | FDA Approved |
| Clinical Trial | NCT03077942 |
| Countries | 1 countries |
Authority Signals
Last Verified7/24/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations2 Validated Nodes