Asciminib – PBPK Modeling Streamlines Development and Avoids Over 10 Clinical Trials

Asciminib (Scemblix®), a novel BCR-ABL tyrosine kinase inhibitor for chronic myeloid leukemia, required establishing safe 80mg and 200mg doses, assessing drug-drug interactions, evaluating food effects, and testing efficacy in patients with liver impairment. The developer used physiologically-based pharmacokinetic (PBPK) modeling to bridge efficacy and safety data across dosing regimens; PBPK predictions informed the drug label, replacing the need for more than 10 traditional clinical pharmacology studies, and Simcyp's ADAM-PBPK simulations showed gastric pH changes would not meaningfully affect absorption. Over 10 clinical pharmacology studies were waived based on the simulations, the FDA approved both dosing regimens using the PBPK data, the modeling expedited the overall development timeline, and the FDA granted accelerated approval in November 2021, with EMA and PMDA endorsements following in 2022.

Certara is a biosimulation software company (Simcyp, Phoenix) whose PK/PD and QSP models predict drug behavior in the body; the FDA itself licenses Certara's software to review sponsor submissions.

This sits in the Build stage of the AI use-case lifecycle: Named examples from the foundation-model labs and platforms pharma teams use to actually construct, train and generate the model or application.