glasdegib glasdegib glasdegib, sold under the brand name daurismo, is a medication for the treatment of newly-diagnosed acute myeloid leukemia (aml) in adults older than 75 years or those who have comorbidities that preclude use of intensive induction chemotherapy. it is taken by mouth and is used in combination with low-dose cytarabine. glasdegibclinical datatrade namesdaurismoother namespf-04449913ahfs/drugs.commonographmedlineplusa619004atc codel01xj03 (who) legal statuslegal status ca: ℞-only us: ℞-only eu: rx-only identifiers iupac name 1--3-(4-cyanophenyl)urea cas number1095173-27-5pubchem cid25166913drugbankdb11978chemspider28518072uniik673dmo5h9keggd10636 yas salt: d11107 ychebichebi:145428chemblchembl2043437echa infocard100.244.738 chemical and physical dataformulac21h22n6omolar mass374.448 g·mol−13d model (jsmol)interactive image smiles cn1ccc(cc1c2=nc3=cc=cc=c3n2)nc(=o)nc4=cc=c(c=c4)c#n inchi inchi=1s/c21h22n6o/c1-27-11-10-16(24-21(28)23-15-8-6-14(13-22)7-9-15)12-19(27)20-25-17-4-2-3-5-18(17)26-20/h2-9,16,19h,10-12h2,1h3,(h,25,26)(h2,23,24,28)/t16-,19-/m1/s1key:sfnsllsynzwzqg-vqimiiecsa-n the recommended dose of glasdegib is 100 mg orally once daily on days 1 to 28 in combination with cytarabine 20 mg subcutaneously twice daily on days 1 to 10 of each 28-day cycle in the absence of unacceptable toxicity or loss of disease control. the most common adverse reactions are anemia, fatigue, hemorrhage, febrile neutropenia, musculoskeletal pain, nausea, edema, thrombocytopenia, dyspnea, decreased appetite, dysgeusia, mucositis, constipation, and rash. it is a small molecule inhibitor of sonic hedgehog, which is a protein overexpressed in many types of cancer. it inhibits the sonic hedgehog receptor smoothened (smo), as do most drugs in its class. history glasdegib was approved for medical use in the united states in december 2018. fda approval was based on a multicenter, open-label, randomized study (bright aml 1003, nct01546038) that included 115 subjects with newly-diagnosed aml who met at least one of the following criteria: a) age 75 years or older, b) severe cardiac disease, c) baseline eastern cooperative oncology group performance status of 2, or d) baseline serum creatinine >1.3 mg/dl. subjects were randomized 2:1 to receive glasdegib, 100 mg daily, with ldac 20 mg subcutaneously twice daily on days 1 to 10 of a 28-day cycle (n=77) or ldac alone (n=38) in 28-day cycles until disease progression or unacceptable toxicity. the trial was conducted in united states, canada and europe. efficacy was established based on an improvement in overall survival (date of randomization to death from any cause). with a median follow-up of 20 months, median survival was 8.3 months (95% ci: 4.4, 12.2) for the glasdegib + ldac arm and 4.3 months (95% ci: 1.9, 5.7) for the ldac alone arm and hr of 0.46 (95% ci: 0.30, 0.71; p=0.0002). glasdegib was granted priority review and orphan drug designation by the u.s. food and drug administration (fda). it was granted orphan drug designation by the european medicines agency (ema) in october 2017. glasdegib was approved for medical use in the european union in june 2020.