- Acute myeloid leukemia
is a cancer of the blood and the bone marrow.
- FDA has
approved the use of Midostaurin drug along with chemotherapy for treating acute
- Patients who took a combination of
midostaurin drug along with chemotherapy lived longer when compared to those
who took chemotherapy alone.
The United States Food
and Drug Administration has recently approved the use of Midostaurin (Rydapt)
drug along with chemotherapy
treating patients with acute myeloid leukemia (AML), with a specific gene
mutation called FLT3 (FMS-like tyrosine kinase 3).
Midostaurin drug has
been approved for use along with a companion diagnostic, LeukoStrat CDx FLT3
Mutation Assay which could detect the FLT3 mutation in patients with AML.
The FDA approval for the
drug was granted to Novartis Pharmaceuticals Corporation, while the approval
for LeukoStrat CDx FLT3 Mutation Assay was granted to Invivoscribe
‘Combination of Midostaurin drug and chemotherapy has been approved by the FDA to treat acute myeloid leukemia.’
Richard Pazdur, M.D.,
acting director of the Office of Hematology and Oncology Products, in the FDA's
Center for Drug Evaluation and Research and director of the FDA's Oncology
Center of Excellence, said, "Rydapt is the first targeted therapy to treat
patients with AML, in combination with chemotherapy."
"The ability to detect
the gene mutation with a diagnostic test means doctors can identify specific
patients who may benefit from this treatment."
Studying the Efficacy
and Safety of Midostaurin Drug
The efficacy and the
safety of Midostaurin drug for patients with acute myeloid leukemia were
studied by conducting a randomized trial on 717 patients who were not
previously treated for acute myeloid leukemia
The findings of the
trial revealed that patients who received a combination treatment of
midostaurin along with chemotherapy were found to live longer than those who
received only chemotherapy.
The patients who
received the combination of Midostaurin along with chemotherapy in the trial
went longer without complications of the disease when compared to the patients
who received chemotherapy alone.
Midostaurin (Rydapt) is
a kinase inhibitor drug which works by blocking several enzymes that could
promote cell growth.
The detection of the
FLT3 mutation in blood or bone marrow samples, using the LeukoStrat CDx FLT3
Mutation Assay, may help the patient to be eligible for treatment in
combination with chemotherapy.
Common side effects of
Midostaurin drug include
- low levels of white blood cells with
(inflammation of the mucous membrane)
- high blood sugar
- upper respiratory tract
Acute Myeloid Leukemia
- Midostaurin drug should not be used
in patients who are allergic to the drug
- Women who are
pregnant or breastfeeding must not take the drug as it could harm the
- Patients with symptoms of lung damage should not use
the drug due to pulmonary toxicity
It is a rapidly
progressing cancer which is formed in the bone marrow and leads to an elevated
white blood cell count in the bloodstream.
The National Cancer
Institute estimated that around 19,930 people were
diagnosed with AML in 2016. And around 10,430 people are projected to die due
to the disease.
Facts on Acute Myeloid
- Acute myeloid leukemia is the
second most common type of cancer in children
- Acute myeloid
leukemia (AML) is common among people who are exposed to large amounts of
radiation and chemicals
- The five-year survival rate for
acute myeloid leukemia ranges from 65-75%
- Around 43,000
people in the United States are affected with leukemia every
- Smoking, genetic disorders and certain blood disorders
like myelodysplatic diseases (chronic bone marrow diseases) are risk factors of
acute myeloid leukemia
- FDA approves new combination treatment for acute
myeloid leukemia - (https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm555778.htm)
- Acute Myeloid Leukemia (AML) - (https://www.stjude.org/disease/acute-myeloid-leukemia.html)
- Leukemia Facts - (https://www.mdanderson.org/cancer-types/leukemia/leukemia-facts.html)