New drug for leukemia! Pfizer's new Hedgehog pathline inhibitor Daurismo has been approved for first-line treatment of acute myelin white blood:

U.S. drug giant Pfizer has approved the target cancer drug Daurismo (glasdegib), combined with low-dose LD-AC chemotherapy, a new first-line treatment, Pfizer announced. Confirmed adult patients with type 2 acute myeloid leukemia (AML), as follows: (1) elderly AML patients aged 75 and over; Previously, the FDA had granted Daurismo priority review eligibility.
the approval, making Daurismo the first and only HEdgehog signaling pathens inhibitor to be approved by the FDA for the treatment of AML, will provide an important treatment option for a group of incurable patients who are unable to receive intensive chemotherapy due to age or other diseases.
note that Daurismo's drug label has a black box warning of embryo-fetal toxicity. In addition, the drug has not been studied in patients with severe kidney damage or moderate to severe liver damage.
Daurismo is an oral Smoothened protein (SMO) inhibitor that inhibits the Hedgehog signaling path, which plays a vital role in the embryo's development by inhibiting the SMO subject. However, in adults, abnormal activation of the Hedgehog signaling path is thought to contribute to the development and survival of cancer stem cells and play a role in the development and development of many types of cancer, including solid tumors and malignant tumors of the blood system.
, Pfizer is currently working on a Phase III BRIGHT clinical program to evaluate Daurismo's combined use with other drugs commonly used to treat AML to explore the full potential of Daurismo's treatment of AML.
Daurismo is based on data from a critical, randomized, open label, multi-center, international Phase II clinical study (BRIGHT 1003). In the study, 115 new AML patients were randomly assigned to Daurismo and LD-AC in a 2:1 ratio or LD-AC alone. More than half (51%, 39) of the 77 patients treated in combination with Daurismo and LD-AC were secondary AML (AML due to previous blood/bone marrow disease or previous cancer therapy). Of these 39 secondary AML patients, 11 were previously treated with low methylated agents, and historical data show that the prognostication of such patients is poor and that treatment options are limited to clinical trials or palliative care.
results showed that the total survival of Daurismo and LD-AC combined treatment groups was significantly extended (medium OS: 8.3 months vs 4.3 months), and the risk of death decreased significantly by 54% (HR-0.46, 95% CI:0.30-0.71, single arm p-0.0002) compared to the LD-AC treatment group.
study, the most common (incident rate ≥20%) adverse reactions reported by the LD-AC treatment group compared to the LD-AC treatment group included anemia (43% vs 42%) and fatigue (43% vs 42%) 36% vs 32%), bleeding (36% vs 42%), fever neutral granulocyte reduction (31% vs 22%), musculoskeletal pain (30% vs 17%), nausea (nausea) 29% vs 12%), edema (30% vs 20%), plateplate reduction (30% vs 27%), breathing difficulties (23% vs 24%), decreased appetite (21%) vs 7%), difficulty swallowing (21% vs 2%), mucositis (21% vs 12%), constipation (20% vs 12%), and rash (20% vs 7%). The most common severe adverse reactions in the Daurismo-LD-AC treatment group (incident rate ≥5%) were a decrease in febrile neutral granulocytes (29%), pneumonia (23%), bleeding (12%), anemia (7%) and sepsis (7%).
Pfizer's global president of oncology, Andy Schmeltz, said Daurismo is the second drug Pfizer has received FDA approval to treat AML in the past 14 months, providing a new treatment for some of the most difficult patients with very limited treatment options to improve their overall survival. In particular, Daurismo will provide a new oral drug for groups of patients who cannot opt for intensive chemotherapy, combined with low-dose chemotherapy, to improve their chances of survival. (Bio Valley)