dm+d

Unassigned

New Medicines

Primary immunodeficiency (WHIM syndrome) in patients aged 13 years and older

Information

New molecular entity
X4 Pharmaceuticals
X4 Pharmaceuticals

Development and Regulatory status

None
None
Phase III Clinical Trials
Yes
Yes

Category

A selective, small molecule antagonist of C-X-C chemokine receptor type 4 (CXCR4) that binds allosterically to the extracellular region of the receptor and inhibits C-X-C motif chemokine 12 stimulation of different intracellular variants of CXCR4.
WHIM syndrome is a rare primary immunodeficiency disorder, which is characterised by warts, hypogammaglobulinemia, infections, and myelokathexis. Individuals with WHIM syndrome are more susceptible to potentially life-threatening bacterial infections [1]. In 2014, it was estimated that there were 0.002 in 10,000 people affected by WHIM syndrome in the European Union [2].
Primary immunodeficiency (WHIM syndrome) in patients aged 13 years and older
Oral

Further information

Yes

Evidence based evaluations