Asciminib

Asciminib, sold under the brand name Scemblix, is a medication used to treat Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML).

[5] The most common adverse reactions include upper respiratory tract infections, musculoskeletal pain, fatigue, nausea, rash, and diarrhea.

[7] In the pooled safety population in participants with newly diagnosed and previously treated Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase, the most common adverse reactions include musculoskeletal pain, rash, fatigue, upper respiratory tract infection, headache, abdominal pain, and diarrhea.

In the wild-type protein, when myristoylated N-terminus binds to the allosteric site, the kinase has reduced activity.

[10] The trial was conducted at 18 sites in ten countries (Australia, France, Germany, Italy, Japan, Netherlands, the Republic of Korea, Singapore, Spain, and the United States).

[10] The efficacy of asciminib in the treatment of participants with Ph+ CML in chronic phase (Ph+ CML-CP), previously treated with two or more tyrosine kinase inhibitors was evaluated in the multi-center, randomized, active-controlled, and open-label study ASCEMBL (NCT 03106779).

[9] A total of 405 participants were randomized (1:1) to receive either asciminib or investigator-selected tyrosine kinase inhibitors (IS-TKIs) (imatinib, nilotinib, dasatinib, or bosutinib).

[7][20][21][22] In July 2024, the US Food and Drug Administration (FDA) granted priority review designation to asciminib for the treatment of newly diagnosed adults with Philadelphia chromosome-positive CML in chronic phase.