FDA clears novel cancer treatments

December 4, 2018

Here are the top 2 novel cancer drugs cleared by the agency.

FDA recently approved 2 novel cancer treatments, including the second “precision medicine” cancer therapy based on a common biomarker across genes.

Here are the top 2 novel cancer drugs cleared by the agency:

1. Larotrectinib (Vitrakvi, Loxo Oncology) treats adult and pediatric patients whose cancers have with solid tumors with a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity and have no satisfactory alternative treatments or that have progressed following treatment.

This is the second time the agency has approved a cancer treatment based on a common biomarker across different types of tumors rather than the location in the body where the tumor originated. “The approval marks a new paradigm in the development of cancer drugs that are ‘tissue agnostic,’” FDA said in a statement.

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"This new site-agnostic oncology therapy isn’t specific to a cancer arising in a particular body organ, such as breast or colon cancer. Its approval reflects advances in the use of biomarkers to guide drug development and the more targeted delivery of medicine,” said FDA Commissioner Scott Gottlieb, MD, in the statement. “We now have the ability to make sure that the right patients get the right treatment at the right time.”

Vitrakvi has shown some benefit with a multitude of solid tumors, with the specific NTRK gene type, Mark Ginestro, principal at KPMG Strategy, told FormularyWatch.

“This essentially has shown a greater degree of sophistication of connecting gene markers with specific medications. For more than a decade, a lot of drugs have been paired with diagnostics or have a biomarker to check to make sure the drug is appropriate. I wouldn’t call this a culmination of a trend, but progress on a path toward personalized medicine,” Ginestro added.

2.  Gilteritinib (Xospata, Astellas Pharma) tablets treats adult patients with relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation as detected by an FDA-approved test.

FDA also approved an expanded indication for a companion diagnostic, LeukoStrat CDx FLT3 Mutation Assay (Invivoscribe Technologies) to detect the FLT3 mutation.

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“Approximately 25% to 30% of patients with AML have a mutation in the FLT3 gene. These mutations are associated with a particularly aggressive form of the disease and a higher risk of relapse,” said Richard Pazdur, MD, director of the 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. “Xospata targets this gene and is the first drug to be approved that can be used alone in treating patients with AML having a FLT3 mutation who have relapsed or who don’t respond to initial treatment.”

The efficiency of Xospata was studied in a clinical trial of 138 patients with relapsed or refractory AML having a confirmed FLT3 mutation. Twenty-one percent of patients achieved complete remission or complete remission with partial hematologic recovery with treatment.

Of the 106 patients who required red blood cell or platelet transfusions at the start of treatment with Xospata, 31% became transfusion-free for at least 56 days.

Read more: Top 3 new FDA approvals