Leukemia is a type of blood cancer that affects many individuals worldwide. Myelogenous leukemia is a specific type of leukemia that originates from bone marrow cells.

It can be categorized into two types: chronic myeloid leukemia (CML) and acute myeloid leukemia (AML). Despite advancements in treatment options, there are still challenges associated with managing the disease. However, there is hope with recent approval of Asciminib by the U.S. Food and Drug Administration (FDA).

What is Asciminib?

Asciminib is a once-daily oral medication created by Novartis that serves as a treatment option for CML.

The drug is a new type of BCR-ABL kinase inhibitor that targets a specific area of the enzyme and blocks the signal pathway that leads to leukemia cell growth. Asciminib received breakthrough therapy designation by the FDA in 2018, which indicates its potential to provide significant clinical benefits in treatment-resistant cases of CML that have not improved with other therapies.

What does the FDA Approval of Asciminib Mean?

The FDA approval of Asciminib is a significant step forward for myelogenous leukemia treatment. According to the FDA, the medication’s approval is based on the results of a phase III clinical trial, ASCEMBL.

The ASCEMBL study’s objective was to determine if Asciminib could improve the major molecular response (MMR) rate in patients with heavily pretreated chronic phase CML. MMR measures the amount of BCR-ABL protein levels in the blood, and it is a crucial factor in evaluating the effectiveness of the treatment in controlling CML.

Participants in the study were randomized to receive either Asciminib or Bosutinib, an FDA-approved medication for CML.

According to the study results, patients receiving Asciminib had a 25.5% MMR rate compared to the 13.2% rate of those receiving bosutinib. This presented a significant increase in the MMR rate of people treated with Asciminib and showcased the effectiveness of this new medication in treating the disease.

Asciminib Side Effects

Like all drugs, Asciminib has side effects that must be considered before using it. The most common side effects reported during the trial were muscle pain, nausea, headache, and diarrhea.

In rare cases, some patients experienced low neutrophil count, anemia, or fatigue. However, these side effects were manageable and did not cause damage or long term problems. In conclusion, the drug showed a reasonable safety profile that is consistent with other drugs in the same category.

Conclusion

The FDA’S approval of Asciminib marks a significant milestone in myelogenous leukemia treatment. It is a promising alternative for patients that are not responding well to other treatments for CML.

The results of the clinical study spearheaded the development of the medication and highlight the potential of Asciminib to improve the MMR rate in patients with heavily pretreated chronic phase CML. While there is a need for further research on its use in other forms of myelogenous leukemia, the FDA’s approval is a giant leap forward in treating the disease.