Marketing Authorization Outside of the EU
Philadelphia chromosome-positive CML-CP, CML-CP
PHILADELPHIA CHROMOSOME-POSITIVE CML, T315I MUTATION, EMA
The United States Food and Drug Administration (FDA) approved marketing authorization for asciminib on October 29, 2021 for 2 indications. First, matching the approval at the EMA, asciminib was approved for the treatment of adult patients with Philadelphia chromosome-positive CML-CP previously treated with 2 or more TKIs. Second, asciminib was approved for the treatment of adult patients with Philadelphia chromosome-positive CML-CP with the T315I mutation. The FDA approval for the Philadelphia positive CML-CP population with the T315I mutation was based on the X2101 study. The daily dose of asciminib in the X2101 study for this subset of patients was 5 times higher than in the ASCEMBL trial. This indication, based on a phase I study and a higher asciminib dosage, was not requested by the company in the initial EMA marketing authorization application. As a result, the FDA approval included this second indication, but it was not included in the EMA approval.