dm+d

Unassigned

New Medicines

Activated phosphoinositide 3-kinase delta syndrome (APDS) in patients aged 12 years and older weighing ≥45kg

Information

New molecular entity
Pharming Group NV
Pharming Group NV

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes

Category

Oral selective PI3Kδ inhibitor, taken twice daily.
Activated PI3K-delta syndrome is a disorder that impairs the immune system. Individuals with this condition often have low numbers of white blood cells (lymphopenia), particularly B cells and T cells. Another possible feature of activated PI3K-delta syndrome is abnormal clumping of white blood cells. The prevalence of activated PI3K-delta syndrome is unknown [1].
Activated phosphoinositide 3-kinase delta syndrome (APDS) in patients aged 12 years and older weighing ≥45kg
Oral