Pharming Group NV has won US regulatory approval for Joenja (leniolisib), a small molecule drug in-licensed from Novartis for the treatment of a rare genetic disorder that impairs the immune system. The disorder is activated phosphoinositide 3-kinase delta syndrome (APDS) which is estimated to affect one to two people per million and can lead to infections in the ears, sinuses and the upper and lower respiratory tracts. Infections usually begin in infancy. Patients with the disease are also susceptible to blood cell cancers like lymphoma, according to the Food and Drug Administration.
