The cell and gene therapy landscape continues to evolve rapidly, with significant advancements in regulatory approvals and clinical development. Several companies have recently achieved key milestones, expanding treatment options for patients with hematological malignancies and solid tumors.
Regulatory Milestones in China
Legend Biotech's ciltacabtagene autoleucel (cilta-cel), marketed as Carvykti, has been approved by China’s National Medical Products Administration (NMPA) for the treatment of adults with relapsed or refractory (r/r) multiple myeloma (MM). This approval targets patients who have previously received at least three lines of therapy, including a proteasome inhibitor and an immunomodulatory agent. Carvykti is a BCMA-directed chimeric antigen receptor T-cell (CAR-T) therapy, offering a new treatment option for patients with limited alternatives.
JW Therapeutics also received approval from China’s NMPA for its supplemental biologics license application for relmacabtagene autoleucel injection (relma-cel), marketed as Carteyva. This approval expands the use of Carteyva to include the treatment of adults with r/r mantle cell lymphoma (MCL). Carteyva is another CAR-T therapy that has demonstrated efficacy in treating hematological malignancies.
Clinical Advancements and New Trials
Galapagos has received clearance from the FDA for its investigational new drug (IND) application for GLPG5101, an investigational CD19-directed CAR-T therapy. This clearance allows Galapagos to initiate a phase 1/2 clinical trial to evaluate GLPG5101 for the treatment of r/r non-Hodgkin lymphoma (NHL). This trial will assess the safety and efficacy of GLPG5101 in patients with relapsed or refractory NHL, potentially providing a new therapeutic option for this patient population.
CARsgen Therapeutics has completed enrollment in its pivotal phase 2 clinical trial (NCT04581473) evaluating satricabtagene autoleucel (satri-cel; CT041). Satri-cel is an investigational CAR-T therapy that targets CLDN18.2, a protein expressed in gastric and gastroesophageal cancers. The trial is focused on patients with CLDN18.2-positive advanced gastric/gastroesophageal (GC/GEJ) cancer. This trial could pave the way for a new targeted therapy for patients with these aggressive cancers.
