Leniolisib (Joenja)

Canada’s Drug Agency (CDA-AMC) recommends that Joenja be reimbursed by public drug plans for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and pediatric patients aged 12 years and older if certain conditions are met. The Canadian Drug Expert Committee (CDEC) determined that it is uncertain whether Joenja demonstrates acceptable clinical value compared with best supportive care (BSC), which includes immunoglobulin (Ig) replacement therapy (IRT), steroids, and immunomodulators, in patients with APDS. Given that Joenja is expected to be an alternative to BSC, acceptable clinical value refers to at least comparable value versus BSC. Evidence from a clinical study showed that Joenja given for 12 weeks likely helps some immune cell populations return to normal and likely helps reduce the size of enlarged lymph nodes when compared with BSC in patients with APDS. Findings from a clinical study where treatment continued for up to 6 years showed that patients receiving Joenja may have fewer infections and use less IRT. Evidence from 3 additional studies supported the findings from the clinical studies showing a possible reduction in infections among patients receiving treatment with Joenja compared to without Joenja. There was uncertainty in the evidence supporting clinical value due to the clinical studies enrolling a small number of patients, the 12-week duration of 1 study, the focus being on end points that were not direct measures of patient-important outcomes, and the possibility of incomplete, inaccurate, or low-quality data used in the 3 supportive studies. CDEC established that there was significant unmet clinical need due to the severity of APDS, despite available treatments, and due to challenges with evidence generation in this patient population. Joenja is the first Health Canada–approved therapy that treats the underlying cause of APDS, which BSC does not. CDEC concluded that Joenja addresses a significant unmet clinical need, despite the uncertainty in the clinical value. Based on all of the preceding considerations, CDEC recommended that Joenja be reimbursed.