HEMATOLOGY

The FDA has accepted a biologics license application for the investigational agent luspatercept for the treatment of adult patients with very low to intermediate-risk myelodysplastic syndromes&ndash;associated anemia who have ring sideroblasts and require red blood cell transfusions, and for the treatment of adult patients with beta-thalassemia&ndash;associated anemia who require RBC transfusions. In addition, a priority review designation was granted for the beta-thalassemia indication.<br /> &nbsp;

According to the pivotal phase III CLL14 study presented during the 2019 American Society of Clinical Oncology Annual Meeting, venetoclax plus obinutuzumab demonstrated a lengthening in progression-free survival time for patients with previously untreated chronic lymphocytic leukemia compared with obinutuzumab plus chlorambucil. Trial results show that the chemotherapy-free combination reduced the risk for disease worsening or death by 65% compared with obinutuzumab plus chlorambucil.

A look back at all the&nbsp;FDA news&nbsp;that happened in the month of&nbsp;May 2019, including several new approvals, orphan drug designations, breakthrough therapy designations, fast track designations, and more.

Copanlisib has been granted with a breakthrough therapy designation from the FDA for the&nbsp;treatment of adult patients with relapsed marginal zone lymphoma who have received at least 2 prior therapies.

A supplemental new drug application has been approved by the FDA&nbsp;to update the label for gilteritinib (Xospata) to include final analysis data from the phase III ADMIRAL trial, which demonstrated an improvement in overall survival with the FLT3 inhibitor compared with salvage chemotherapy in adult patients with relapsed/refractory <em>FLT3</em>-mutant AML.

John M. Burke, MD, reviews the treatment options he considers when treating a patient with chronic lymphocytic leukemia and the factors that sway his clinical decisions.

The R²&nbsp;regimen of lenalidomide (Revlimid) plus rituximab (Rituxan) has been approved by the FDA for the treatment of patients&nbsp;with previously treated follicular lymphoma and marginal zone lymphoma, based on findings from the&nbsp;phase III AUGMENT trial.

Chronic Myeloid Leukemia

Ruxolitinib (Jakafi) has received approval from the FDA as a treatment for&nbsp;adult and pediatric patients &ge;12 years of age with&nbsp;steroid-refractory acute graft-versus-host disease.

The combination of venetoclax (Venclexta) and obinutuzumab (Gazyva) has been approved by the FDA&nbsp;for the frontline treatment of patients with chronic lymphocytic leukemia or small lymphocytic lymphoma, based on findings from the&nbsp;phase III CLL14 trial.

In an 8-3 vote, the FDA&rsquo;s Oncologic Drugs Advisory Committee has recommended against approving a new drug application for quizartinib for adult patients with relapsed/refractory&nbsp;FLT3-ITD&ndash;positive acute myeloid leukemia. The FDA is now scheduled to make a final decision on the application by&nbsp;August 25, 2019.

William G. Wierda, MD, PhD, discusses the take home message from the phase III MURANO trial that identified venetoclax plus rituximab as a new treatment option for patients with relapsed/refractory chronic lymphocytic leukemia.

Jacqueline C. Barrientos, MD, MS,&nbsp;discusses the emerging treatment options for patients with CLL, as well as some of the other strategies being looked at in clinical trials.

Alexander E. Perl, MD, discusses the latest advancements in the treatment landscape for patients with acute myeloid leukemia. These advances are driven by 2 factors: a better understanding of the biology of the disease and improving therapeutics to meet that understanding.