Ben Jackson's questions to Pharming Group (PHAR) leadership • Q2 2025
Question
Ben Jackson sought more detail on the specific bottlenecks affecting the rate of VUS patient uptake in the second half of the year. Additionally, he asked about the potential pathway and timeline for a label expansion for Joenja based on the newly identified, broader clinical phenotype for APDS.
Answer
CEO Fabrice Chouraqui explained the VUS reclassification bottleneck is the time required for genetic labs to process new data, identify relevant patients, and for doctors to be informed, which will happen gradually. Chief Medical Officer Anurag Relan clarified that since the new phenotypes are considered APDS, these patients are likely covered by the existing label, potentially avoiding the need for new clinical trials for treatment access.