FDA Approves New Treatment for Certain Patients with Acute Myeloid Leukemia

The FDA today granted approval to Pfizer’s glasdegib (Daurismo) for certain patients with newly-diagnosed acute myeloid leukemia (AML), according to a press release.

Glasdegib, a once-daily oral medicine, is indicated for use in combination with low-dose cytarabine (LDAC), a type of chemotherapy, for adults with AML who are 75 years of age or older or who have other chronic health conditions that may preclude the use of intensive chemotherapy.

The agency also today approved

venetoclax (Venclexta, Genentech and AbbVie) for the treatment of AML.

The standard of care for AML, a rapidly progressing bone marrow cancer with poor survival rates, is typically intensive chemotherapy. However, intensive treatment may not be an option for certain patients, which significantly worsens prognosis. Glasdegib is the first and only FDA-approved Hedgehog pathway inhibitor for AML.

The approval is based on data from the phase 2 BRIGHT 1003 trial, which included 115 patients with newly-diagnosed AML. Patients received either glasdegib plus LDAC or LDAC alone. Of the 77 patients treated with glasdegib plus LDAC, 51% with secondary AML received prior treatment with a hypomethylating agent. According to the data, the median OS was 8.3 months for patients treated with glasdegib plus LDAC compared with 4.3 months for patients treated with LDAC only.

The study included patients with cardiac disease or mild to moderate kidney disease who are often excluded from clinical trials.

Glasdegib comes with a boxed warning to advise health care professionals and patients about the risk of embryo-fetal death or severe birth defects, according to the FDA. The boxed warning also advises men of the potential risk of drug exposure through semen.

The most common adverse effects associated with glasdegib in the trial include low red blood cell count, tiredness, bleeding, febrile neutropenia, muscle pain, nausea, edema, thrombocytopenia, shortness of breath, decreased appetite, distorted taste, pain or sores in the mouth or throat, constipation, and rash.

“We are delighted by today’s approval of Daurismo by the FDA, and are working to gain greater understanding of its role in treating patients with acute myeloid leukemia,” Mace Rothenberg, MD, chief development officer, oncology, Pfizer Global Product Development, said in a statement.

Phase 3 trials are currently evaluating glasdegib in combination with other agents commonly used to treat patients with AML, according to the press release.

Reference

US FDA Approves Daurismo (glasdegib) for Adult Patients with Newly-Diagnosed Acute Myeloid Leukemia (AML) for Whom Intensive Chemotherapy is Not An Option [news release]. Pfizer’s website. https://investors.pfizer.com/investor-news/press-release-details/2018/US-FDA-Approves-DAURISMO-glasdegib-for-Adult-Patients-with-Newly-Diagnosed-Acute-Myeloid-Leukemia-AML-for-Whom-Intensive-Chemotherapy-is-Not-an-Option/default.aspx. Accessed November 21, 2018.