FDA approves novel leukemia, hepatitis C drugs

FDA approved the first treatment for patients with a certain type of acute myeloid leukemia, as well as 2 new drugs to treat hepatitis C.

Vyxeos (Jazz Pharmaceuticals), a fixed combination of chemotherapy drugs daunorubicin and cytarabine. was approved to treat adults with two types of acute myeloid leukemia (AML): newly diagnosed therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC).

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“This is the first approved treatment specifically for patients with certain types of high-risk AML,” said Richard Pazdur, MD, director of FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in a statement from FDA. “Vyxeos combines two commonly-used chemotherapies into a single formulation that may help some patients live longer than if they were to receive the two therapies separately.”

Meanwhile, FDA also approved Vosevi (Gilead Sciences) to treat adults with chronic hepatitis C virus (HCV) genotypes 1-6 without cirrhosis (liver disease) or with mild cirrhosis.

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Vosevi is a fixed-dose, combination tablet containing two previously approved drugs – sofosbuvir and velpatasvir – and a new drug, voxilaprevir. Vosevi is the first treatment approved for patients who have been previously treated with the direct-acting antiviral drug sofosbuvir or other drugs for HCV that inhibit a protein called NS5A.

“Direct-acting antiviral drugs prevent the virus from multiplying and often cure HCV. Vosevi provides a treatment option for some patients who were not successfully treated with other HCV drugs in the past,” said Edward Cox, MD, director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research, in a FDA statement.

The results of two phase 3 clinical trials demonstrated that 96 to 97% of patients who received Vosevi had no virus detected in the blood 12 weeks after finishing treatment, suggesting that patients’ infection had been cured.

FDA also approved glecaprevir and pibrentasvir (Mavyret, AbbVie) to treat adults with chronic hepatitis C virus (HCV) genotypes 1-6 without cirrhosis (liver disease) or with mild cirrhosis, including patients with moderate to severe kidney disease and those who are on dialysis.

The treatment is also approved for adult patients with HCV genotype 1 infection who have been previously treated with a regimen either containing an NS5A inhibitor or an NS3/4A protease inhibitor but not both.

Mavyret is the first treatment of 8 weeks duration approved for all HCV genotypes 1-6 in adult patients without cirrhosis who have not been previously treated. Standard treatment length was previously 12 weeks or more.

“This approval provides a shorter treatment duration for many patients, and also a treatment option for certain patients with genotype 1 infection, the most common HCV genotype in the United States, who were not successfully treated with other direct-acting antiviral treatments in the past,” Cox said in a statement from FDA.

The safety and efficacy of Mavyret were evaluated during clinical trials with approximately 2,300 adults with genotype 1, 2, 3, 4, 5 or 6 HCV infection without cirrhosis or with mild cirrhosis. The trials demonstrated that 92% to100% of patients who received Mavyret for 8, 12 or 16 weeks duration had no virus detected in the blood 12 weeks after finishing treatment, suggesting that patients’ infection had been cured. 

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