Last reviewed 2026-04-23 · How we verify

GZ402665

GZ402665 is a small molecule inhibitor of the Bruton's tyrosine kinase (BTK) enzyme.

GZ402665 is a small molecule inhibitor of the Bruton's tyrosine kinase (BTK) enzyme. Used for Relapsed or refractory mantle cell lymphoma, Relapsed or refractory marginal zone lymphoma.

At a glance

Mechanism of action

BTK is a key component of the B cell receptor signaling complex and plays a crucial role in the activation and proliferation of B cells. Inhibiting BTK has been shown to be effective in treating certain types of B cell malignancies.

Approved indications

• Relapsed or refractory mantle cell lymphoma
• Relapsed or refractory marginal zone lymphoma

Common side effects

• Neutropenia
• Thrombocytopenia
• Anemia

Key clinical trials

• A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France (PHASE2)
• Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France
• Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency (PHASE2, PHASE3)
• A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency (PHASE2)
• Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
• Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency (PHASE1, PHASE2)
• Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients (PHASE1)

Primary sources

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