Module 9 2021

19/03/2021

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 1 st 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

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The Organisation for Professionals in Regulatory Affairs

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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;

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The Organisation for Professionals in Regulatory Affairs

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