Efficacy

Efficacy of Rydapt (Midostaurin) in Leukemia

Rydapt, known generically as midostaurin, is a kinase inhibitor approved by the U.S. Food and Drug Administration (FDA) for the treatment of acute myeloid leukemia (AML) that is FLT3 mutation-positive, as detected by an FDA-approved test. It is used in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation. The efficacy of Rydapt in the treatment of AML was demonstrated in a pivotal phase III clinical trial (RATIFY), which showed a significant improvement in overall survival when midostaurin was added to standard chemotherapy, compared to chemotherapy alone. Patients treated with Rydapt had a median overall survival of 74.7 months compared to 25.6 months for those who received placebo.

Furthermore, the addition of Rydapt to standard chemotherapy has been shown to improve event-free survival, which is the length of time during and after treatment that a patient lives with the disease but it does not get worse. The RATIFY trial reported an event-free survival of 8.2 months for patients receiving midostaurin versus 3.0 months for those on placebo. These results indicate that Rydapt is effective in prolonging both the overall and event-free survival of patients with FLT3-mutated AML when used in conjunction with standard chemotherapy regimens.

Efficacy of Brukinsa (Zanubrutinib) in Leukemia

Brukinsa, or zanubrutinib, is a Bruton's tyrosine kinase (BTK) inhibitor that has shown efficacy in the treatment of mantle cell lymphoma (MCL), a type of leukemia. While its primary approval is for MCL after at least one prior therapy, ongoing studies are assessing its efficacy in other types of B-cell malignancies. In clinical trials, zanubrutinib has demonstrated a high overall response rate in patients with relapsed or refractory MCL. For instance, in a pivotal phase II trial, the overall response rate was reported to be 84%, with a complete response rate of 59%.

While the above data is promising, it is important to note that the efficacy of Brukinsa specifically in the treatment of other forms of leukemia is still being investigated. The drug's mechanism of action, targeting BTK, is relevant to the pathways involved in several leukemias, and ongoing clinical trials are essential to establish its safety and efficacy in these additional contexts. As research progresses, Brukinsa may potentially be used for a broader range of leukemias, but currently, its use is primarily focused on MCL, a distinct subtype of leukemia.