FDA Approves Pfizer’s Acute Myeloid Leukemia Treatment for High-Risk Patients

According to a publication from MarketScreener, pharmaceutical titan Pfizer Inc. has gained FDA approval for Daurismo, a once-a-day oral medication designed to be paired with low-dose cytarabine (a type of chemotherapy) in Acute Myeloid Leukemia patients 75 years and older, or with comorbid conditions.

Acute Myeloid Leukemia

Acute Myeloid Leukemia (or AML) is an aggressive form of leukemia with poor survival rates.

AML starts in the patient’s bone marrow, but due to its aggressive nature it can quickly enter the blood stream. Occasionally it may even spread to other parts of the body, like the lymph nodes, liver, or testicles.

Progression is often rapid, and the preferred course of treatment continues to be intense chemotherapy. Chemotherapy is physically demanding, however, and may not be a viable choice for the elderly or people with comorbid conditions. For these patients, the prognosis can be especially grim.

FDA Approves Daurismo

The FDA’s recent approval of Daurismo hopefully represents a new chapter in treating these especially serious cases.

Daurismo’s randomized Phase 2 clinical trial involved 115 newly-diagnosed AML patients from around the world. One-third of the group (38 patients) received only low-dose cytarabine (LDAC), the other two-thirds (77 patients) received a combination of LDAC therapy and a daily prescribed dosage of Daurismo.

The study found that patients treated with the combined LDAC and Daurismo regimen had a median overall survival of 8.3 months. Patients treated with LDAC alone had a significantly lower median life expectancy at just 4.3 months. That’s a 54% reduction in a patient’s risk of death.

Daurismo and the associated LDAC therapy are not without risk, however. Daurismo has a warning included in its box cautioning of embryo-fetal toxicity – it can cause fetal death or severe birth defects when administered to a woman who’s pregnant. Many patients reported serious averse reactions: 29% of the LDAC and Daurismo group experienced febrile neutropenia (fever stemming from low white-cell count), 23% developed pneumonia, and 12% hemorrhages. That said, these averse reactions are all commonly experienced in chemotherapy patients.

During the first 90 days, patients taking Daurismo and LDAC did report certain adverse affects at significantly higher rates than patients prescribed only LDAC. They were 13% more likely to have musculoskeletal pain, 17% more likely to feel nauseous, and three times as likely to experience a decrease in appetite.

Daurismo is a Hedgehog signaling pathway inhibitor. The Hedgehog signaling pathway is a sort of communication system used by your body for differentiating your cells when you were an embryo. However, in adults, abnormal activation of the Hedgehog pathway is believed to contribute to the formation of cancer cells. Daurismo combats acute myeloid leukemia by suppressing it at the pathway.

Trials Set To Continue

Despite the approval in a select number of patients, Pfizer is continuing to run Phase 3 trials for Daurismo. These trials involve trying different combinations of Daurismo with other common AML treatments in order to determine how to maximize its effectiveness.


Symptoms experienced by chemotherapy patients can often be severe. Do you consider the risks of Daurismo to be a serious deterrent to its reported effectiveness? Share your thoughts with the Patient Worthy Community!

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