The FDA has accepted Pfizer’s New Drug Application (NDA) and granted priority review designation for glasdegib, which is indicted for adult patients with previously untreated acute myeloid leukemia (AML), according to a Pfizer press release.
 
Glasdegib is an investigational oral smoothened inhibitor being evaluated for the treatment of AML in combination with low-dose cytarabine (LDAC), a type of chemotherapy. 
 
Despite recent advancements in treatment, only approximately 1 in 4 patients with AML survive longer than 5 years, according to the press release. AML accounts for approximately 80% of all cases of acute leukemia.
 
“Patients with acute myeloid leukemia who are ineligible for intensive chemotherapy are in critical need of new treatment options to improve their overall survival,” Mace Rothenberg, MD, chief development officer of oncology, Pfizer Global Product Development, said in a press release.
 
According to Pfizer, the submission is based on data from a phase 2 clinical trial evaluating glasdegib combined with LDAC compared with LDAC alone. The trial included 132 patients with previously untreated AML or high-risk myelodysplastic syndrome (MDS) who were not eligible for intensive chemotherapy.
 
Data from the trial demonstrated a significant improvement in the primary endpoint of overall survival (OS). Patients treated with glasdegib plus LDAC had a median OS of 8.8 months compared with 4.9 months for patients treated with LDAC only, representing a 49.9% reduction in the risk of death.
 
According to the press release, the most common adverse effects associated with the treatment were anemia, febrile neutropenia, nausea, decreased appetite, fatigue, and thrombocytopenia.
 
The phase 3 BRIGHT AML 1019 trial, which is evaluating the addition of glasdegib to intensive or non-intensive chemotherapy in patients with diagnosed AML, began enrolling earlier this year. According to Pfizer, the Prescription Drug User Fee Act goal date for decision by the FDA is in December 2018.
 
Reference
 
US FDA Grants Priority Review for Pfizer’s New Drug Application for Glasdegib in Patients with Previously Untreated Acute Myeloid Leukemia [news release]. Pfizer’s website. https://www.pfizer.com/news/press-release/press-release-detail/u_s_fda_grants_priority_review_for_pfizer_s_new_drug_application_for_glasdegib_in_patients_with_previously_untreated_acute_myeloid_leukemia. Accessed June 28, 2018.