AML

Ivosidenib (Tibsovo) has been granted approval by the FDA for the treatment of&nbsp;adult patients with relapsed/refractory <em>IDH1</em>-mutant acute myeloid leukemia.

The FDA has received a supplemental New Drug Application for venetoclax in combination with&nbsp;a hypomethylating agent or low-dose cytarabine as a frontline treatment for&nbsp;patients with acute myeloid leukemia who are ineligible for intensive chemotherapy, Genentech, the manufacturer of the BCL-2 inhibitor, has announced.

Romyelocel-L (human myeloid progenitor cells) has been granted a Regenerative Medicine Advanced Therapy designation by the FDA for the prevention of bacterial and fungal infections in patients with acute myeloid leukemia undergoing induction chemotherapy, according to Cellerant Therapeutics, Inc, the manufacturer of the therapy. RMAT designation holds similar advantages to breakthrough therapy designation.

Treating AML With Myelodysplasia-Related Changes

Treatment Options and Care for Patients Who Develop Therapy-Related AML

CPX-351, a fixed-combination of daunorubicin and cytarabine, has been recommended for approval by the&nbsp;European Medicines Agency&rsquo;s Committee for Medicinal Products for Human Use for the treatment of adult patients with newly diagnosed therapy-related acute myeloid leukemia or AML with myelodysplasia-related changes.

The FDA approved several indications in the month of June, including venetoclax (Venclexta) in chronic lymphocytic leukemia (CLL) and small lymphocytic leukemia (SLL), the combination of binimetinib (Mektovi) plus encorafenib (Braftovi) in melanoma, and bevacizumab (Avastin) in ovarian cancer. The FDA also accelerated approvals for pembrolizumab (Keytruda) in cervical cancer and in primary mediastinal large B-cell lymphoma, while also granting a priority review to glasdegib for acute myeloid leukemia.

Based on data from the phase II BRIGHT 1003 study, the FDA has granted a priority review to a new drug application for glasdegib for use in combination with chemotherapy for the frontline treatment of patients with acute myeloid leukemia, according to Pfizer, the developer of the investigational oral smoothened inhibitor.

William B. Donnellan, MD, was recently promoted to director of Leukemia/Myelodysplastic Syndrome Research at the Sarah Cannon Research Institute in Nashville, Tennessee. Donnellan has served as an investigator of hematologic malignancies since 2014 at the Sarah Cannon Research Institute.

Michael R. Grunwald, MD,&nbsp;discusses new and emerging agents in AML and ALL, as well as sequencing and toxicity challenges with these treatments.<br /> &nbsp;

Alexander Perl, MD, an associate professor of medicine at the University of Pennsylvania, discusses the current challenges with CAR T-cell therapy for the treatment of patients with acute myeloid leukemia

An overall response rate of&nbsp;41.6% (95% CI, 32.9-50.8) was induced with the use of ivosidenib in patients with&nbsp;<em>IDH1</em>-positive relapsed/refractory acute myeloid leukemia, according to data presented at the 2018 ASCO Annual Meeting&nbsp;and published simultaneously online in the <em>New England Journal of Medicine</em>.

In an ideal world, researchers conduct preclinical studies that generate a targeted therapy, which eventually makes its way through early, middle, and late-stage trial development and FDA approval. That smooth transition does not happen often, but early results involving an agent that affects 2 endogenous inhibitors of p53 look promising.

According to results presented at the 2018 ASCO Annual Meeting,&nbsp;moxetumomab pasudotox, a first-in-class recombinant immunotoxin targeting CD22, demonstrated deep and durable responses in a substantial proportion of pretreated patients with relapsed/refractory hairy cell leukemia.

Based on data from the ongoing phase III ADMIRAL study, a new drug application for&nbsp;gilteritinib has been granted a priority review by the FDA for&nbsp;the treatment of adult patients with <em>FLT3</em> mutation&ndash;positive relapsed or refractory acute myeloid leukemia,&nbsp;according to Astellas Pharma, the manufacturer of the FLT3 inhibitor.

The combination of prexigebersen, a Liposomal Grb2 Antisense oligodeoxynucleotide, and low-dose cytarabine was associated with an overall response rate of 47% in elderly patients with acute myeloid leukemia, according to findings from an ongoing phase II trial.