Experts at the 2025 ICE-T Symposium reviewed CAR T-cell therapy, bispecific antibodies, and gene therapy across myeloma, lymphoma, autoimmune disease, and solid tumors, emphasizing safety and access. Although ICANS remains the dominant paradigm, clinicians must now recognize a second category of neurologic complications characterized by delayed onset, movement disorders, and prolonged recovery. The observed incidence of approximately 14% suggests that these events are not rare and may become increasingly relevant as CAR T-cell therapy is deployed earlier in the disease course and in larger patient populations. Patients with SLE and related disorders consistently experience lower rates of severe CRS and minimal neurotoxicity. However, long-term efficacy and safety remain under study. This difference may reflect lower disease burden, distinct immune environments, or differences in CAR T expansion kinetics. Although hypogammaglobulinemia and transient cytopenias remain common, these effects are generally manageable and limited in duration. The median duration of response was also prolonged (14.8 vs 9.7 months), indicating more sustained disease control. Although OS analysis in the intention-to-treat population was affected by crossover in approximately 56% of patients in the standard arm, the median OS favored ide-cel (41.4 vs 37.9 months), and adjusted analyses further supported a survival benefit (HR, 0.72). Collectively, these findings reinforce the movement of CAR T-cell therapy into earlier lines of treatment. Next-generation CAR T platforms are now focused on improving both efficacy and safety profiles. The emphasis is not solely on growth, but on building a sustainable and scalable model that can support the next generation of cellular therapies. CAR T-cell therapy has produced survival outcomes in relapsed/refractory multiple myeloma and aggressive lymphomas that were unimaginable a decade ago. Those outcomes remain largely inaccessible to patients who do not live near a FACT-accredited academic center, whose disease does not allow the weeks required for manufacturing, or whose geography or support system cannot meet the demands of the referral pathway.

EU approval makes Joenja the first and only approved APDS treatment across the EU, Norway, Iceland and Liechtenstein, with Germany launch due in Q3 2026. Leniolisib is also being evaluated in two Phase II clinical trials in primary immunodeficiencies (PIDs) with immune dysregulation. The safety and efficacy of leniolisib has not been established for PIDs with immune dysregulation beyond APDS. About Pharming Group N.V. Pharming Group N.V. (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. The EU approval advances Pharming’s global rare disease expansion strategy for Joenja. According to Pharming, Joenja already has approvals in the United States, United Kingdom, Japan, Australia and Israel, and the new European authorization further broadens its international treatment footprint for APDS. ... May 7, 2026 Pharming Group announces presentations at CIS 2026 Annual Meeting, including leniolisib pediatric data in APDS and clinical experience in CVID and related disorders Pharming (PHARM) received European Commission marketing authorization for Joenja (leniolisib), the first and only approved treatment in the EU for activated PI3K delta syndrome (APDS) in patients aged 12 and older. First EU launch is expected in Germany in Q3 2026, with further roll-out after national reimbursement decisions. The approval, based on a Phase II/III trial and long-term extension data, is valid in all 27 EU states plus Norway, Iceland and Liechtenstein, and supports Pharming’s rare disease expansion alongside existing approvals in the US, UK, Japan, Australia and Israel. Investors watch these conditions because they create steady, long‑term demand for diagnostics and specialized treatments (including replacement therapies and gene therapies); successful clinical trials or approvals can rapidly change a company’s revenue outlook and valuation, similar to discovering a cure for a chronic condition that many patients need.