FDA Recently Approved Three Drugs to Treat Leukemia

Acute myeloid leukemia (AML) is a rapidly progressing cancer that forms in the bone marrow and results in an increased number of white blood cells in the bloodstream. The National Cancer Institute of the National Institutes of Health estimates that approximately 21,380 people will be diagnosed with AML this year and that 10,590 patients with AML will die of the disease. it is celebrated that the U.S. Food and Drug Administration recently approved three drugs to treat leukemia.

  1. Gemtuzumab Ozogamicin

On September 1, 2017, the U.S. Food and Drug Administration approved Mylotarg® (gemtuzumab ozogamicin) for the treatment of adults with newly diagnosed acute myeloid leukemia (AML). Additionally, the agency approved the antibody-drug conjugate for the treatment of patients aged 2 years and older with CD33-positive relapsed/refractory AML.

Gemtuzumab ozogamicin is a recombinant, humanized anti-CD33 monoclonal antibody covalently attached to the cytotoxic antitumor antibiotic calicheamicin (N-acetyl-γ-calicheamicin) via a bifunctional linker (4-(4-acetylphenoxy)butanoic acid). Calicheamicins are potent anti-tumour antibiotics that were initially identified by their ability to damage DNA in screening tests. In this antibody-drug-conjugate, the antibody binds to and is internalized by tumor cells expressing CD33 antigen (a sialic acid-dependent glycoprotein commonly found on the surface of leukemic blasts), thereby delivering the attached calicheamicin to CD33-expressing tumor cells.

In May 2000, FDA approved MylotargTM as a stand-alone treatment for older patients with CD33-positive AML who had experienced a relapse. Subsequent confirmatory trials failed to verify clinical benefit and demonstrated safety concerns which leaded to that Mylotarg® was voluntarily withdrawn from the market. Now the approval includes a lower recommended dose, a different schedule in combination with chemotherapy or on its own, and a new patient population.

  1. Enasidenib

Enasidenib (AG-221)

IDHIFA® (enasidenib), studied by Celgene Corporation and Agios Pharmaceuticals, Inc., was granted approval from the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory AML with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA approved test. This is the first FDA approval for relapsed or refractory AML specifically with an IDH2 mutation, which represents between 8 and 19 percent of AML patients.

The most common adverse reactions occurring in greater than 20% of patients were nausea, vomiting, diarrhea, elevated bilirubin, and decreased appetite. Differentiation syndrome occurred in 14% of patients. Women who are pregnant or breastfeeding should not take Idhifa because it may cause harm to a developing fetus or a newborn baby.

  1. Midostaurin

On April 28, 2017, the U.S. Food and Drug Administration today approved Rydapt® (midostaurin, formerly PKC412, Novartis Pharmaceuticals Corp.) for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) who are FMS-like tyrosine kinase 3 mutation-positive (FLT3+), as detected by an FDA-approved test, in combination with chemotherapy. Rydapt® is also approved to treat adults with advanced systemic mastocytosis (SM), which includes aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN) and mast cell leukemia.

Common sides effects reported during Rydapt® treatment for AML included low level of white blood cells with fever (febrile neutropenia); nausea; redness, pain or ulcers inside the mouth (mucositis); vomiting; headache; bruising; muscle or bone pain; nose bleeds; device-related infection; high blood sugar levels (hyperglycemia) and upper respiratory infections. Common side effects reported during treatment for ASM, SH-AHM or mast cell leukemia included nausea; vomiting; diarrhea; swelling of the hands, feet or ankles; muscle or bone pain; stomach-area pain; tiredness; upper respiratory infection; constipation; fever; headache and trouble breathing.


FDA approves Mylotarg for treatment of acute myeloid leukemia

FDA granted regular approval to enasidenib for the treatment of relapsed or refractory AML

FDA approves new combination treatment for acute myeloid leukemia

Novartis receives FDA approval for Rydapt® in newly diagnosed FLT3-mutated acute myeloid leukemia (AML) and three types of systemic mastocytosis (SM)