Two medications, Daurismo and Venclexta, have passed the final rounds of review by the US Food and Drug Administration (FDA) and been approved for patients with acute myeloid leukemia (AML). These drugs are intended for use in patients who are not candidates for intensive chemotherapy. Intensive chemotherapy is not recommended for patients over the age of 75 or those with certain health conditions due to the severe side effects that it causes.
Daurismo, marketed by Pfizer, was tested in clinical trials in combination with a low-dose of the chemotherapy drug, cytarabine. Known as a hedgehog inhibitor, Daurismo is a targeted medicine that interferes with the hedgehog-signaling pathway. The hedgehog-signaling pathway is involved in cell differentiation and growth, and problems in this pathway can cause out-of-control cell growth and lead to certain types of cancers, such as AML. Daurismo was tested in a randomized clinical trial of approximately 100 people. Patients who received Daurismo in addition to cytarabine lived an average of four months longer than those who only received the chemotherapy.
The drug Venclexta is already available to patients with chronic lymphocytic leukemia, but the FDA expanded its approved indications to include AML after two non-randomized clinical trials. These trials measured the number of AML patients who went into complete remission, meaning there were no traces of cancer in the body, and how long they stayed in remission.
In the first trial, 37 percent of patients achieved complete remission after receiving Venclexta along with the chemotherapy drug azacitidine and stayed in remission for an average of 5.5 months. In the same study, 54 percent of patients who received Venclexta and decitabine, another chemotherapy drug, went into complete remission that lasted for an average of 4.7 months. In the other trial, 21 percent of patients achieved complete remission for an average of six months when Venclexta was used with cytarabine. Venclexta was developed by AbbVie and is marketed by AbbVie and Genentech USA Inc (Roche).
Both medications were reviewed under special FDA procedures that are used to speed up the approval process for medicines to treat serious illnesses without adequate alternatives. They were also granted orphan drug designation, which provides modest financial incentives to companies to encourage the development of drugs for rare diseases.