Activated Phosphoinositide 3-kinase Delta Syndrome (“APDS”)
Activated Phosphoinositide 3-kinase Delta Syndrome (“APDS”) is an ultra-rare, inherited, debilitating disease that impairs the immune system.
APDS is a primary immunodeficiency (PID) where white blood cells in the immune system don’t work correctly, particularly ones that are called B cells and T cells. Normally, these cells recognise and attack viruses and bacteria to prevent infection. In APDS, the white blood cells are abnormal so these patients often have frequent infections, particularly in the airways and lungs, which in time can lead to a condition called bronchiectasis. This long-term condition damages the passages leading from the windpipe to the lungs (bronchi) and can cause significant breathing problems. Furthermore, individuals can be at greater risk of developing conditions linked with the over production of white blood cells (such as swollen lymph nodes and lymphoma). APDS affects approximately 1-2 per million globally and there is currently no specific pharmacologic treatment available, instead treatment options are limited to symptom reduction.
APDS is a disorder that impairs the immune system. Individuals with the condition often have low levels of white blood cells, which normally recognize and attack foreign invaders, such as viruses and bacteria, to prevent infection. Beginning in childhood, people with APDS develop recurrent infections, particularly in the lungs, sinuses, and ears. Over time, recurrent respiratory tract infections can lead to a condition called bronchiectasis, which damages the passages leading from the windpipe to the lungs (bronchi) and can cause breathing problems. APDS affects approximately 1-2 per million globally and there is currently no approved treatment available.
Pharming acquired the exclusive global license to CDZ173 (leniolisib), a late-stage drug for the treatment of APDS, from Novartis in 2019. Leniolisib is currently being studied in a registration-enabling Phase II/III clinical trial. To date, leniolisib has proven to be safe and well tolerated in healthy subjects as well as effective in limiting disease impact in APDS patients during the Phase I, first in human trial and the ongoing open label extension trial¹.
If approved, leniolisib is expected to reach the market in mid-2022. Pharming will then commercialise leniolisib through its existing commercial infrastructure in the US and Europe and look for ways to make the drug available in other markets worldwide.
- V. Koneti Rao, et al, 2017. Effective “activated PI3Kd syndrome”– targeted therapy with the PI3Kd inhibitor leniolisib. Blood. 2017;130(21):2307-2316