Module 9 2024

03/09/2024

Kymriah: Safety and Risk Minimisation Measures

• Cytokine release syndrome (CRS) reported in 77% of patients (47% Grade 3 or 4) • 1 patient died as a direct result of CRS; 1 patient died had resolving CRS and other complications • Usually observed within 1st 8 weeks post-infusion • Adverse events considered manageable with appropriate risk minimisation measures • Risk minimisation measures include: • Non-interventional post-authorisation safety study (PASS) • Post-authorisation efficacy studies (PAES) • Report on real world evidence of Kymriah in children below the age of 3 years with B ALL • Observational study in DLBCL (Category 1) Follow up reports for pivotal study C2201 (5 years follow-up) • Additional study evaluating efficacy and safety of Kymriah in adult patients with relapsed or refractory B-cell aggressive NHL

The Organisation for Professionals in Regulatory Affairs

Kymriah European Assessment Report 28 th June 2018

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Learning Outcomes: Recap

• Understand aspects of clinical development for biopharmaceuticals;

• Identify key considerations when designing a first-in-human study;

• Be aware of the clinical pharmacology of biopharmaceuticals;

• Appreciate the continuum of clinical development post first-in-human studies;

• Recognise the continuation of clinical development post-approval

The Organisation for Professionals in Regulatory Affairs

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