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Personalized Medicine's Slice of the Pie

For the fourth year running, personalized medicines have made up more than 20 percent of new drugs approved by the US Food and Drug Administration, according to a new report from the Personalized Medicine Coalition.

In all, the report says the agency approved 19 personalized medicines — including three gene therapies — in 2017, noting that's the highest number of personalized medicines to garner approval in a year. These newly approved therapeutics include Novartis' Kisqali (ribociclib), Neurocrine Biosciences' Ingrezza (valbenazine), and AbbVie's Mavyret (glecaprevir and pibrentasvir). GenomeWeb's Turna Ray likewise has reported that FDA approved 19 personalized medicines in 2017, as compared to 14 in 2016 and about a dozen in 2015.

PMC also notes that the FDA approved a number of new indications for already-existing drugs, such as Merck's Keytruda (pembrolizumab), Bristol-Myers Squibb's Opdivo (nivolumab), and Novartis' Tasigna (nilotinib), among others. It says the expanded indication of Keytruda "is particularly significant" since it's the first time that a tissue-agnostic cancer drug has been approved.

This boost in approvals, PMC President Edward Abrahams says in a statement, suggests "FDA is increasingly committed to supporting" targeted therapies.

The Scan

Positive Framing of Genetic Studies Can Spark Mistrust Among Underrepresented Groups

Researchers in Human Genetics and Genomics Advances report that how researchers describe genomic studies may alienate potential participants.

Small Study of Gene Editing to Treat Sickle Cell Disease

In a Novartis-sponsored study in the New England Journal of Medicine, researchers found that a CRISPR-Cas9-based treatment targeting promoters of genes encoding fetal hemoglobin could reduce disease symptoms.

Gut Microbiome Changes Appear in Infants Before They Develop Eczema, Study Finds

Researchers report in mSystems that infants experienced an enrichment in Clostridium sensu stricto 1 and Finegoldia and a depletion of Bacteroides before developing eczema.

Acute Myeloid Leukemia Treatment Specificity Enhanced With Stem Cell Editing

A study in Nature suggests epitope editing in donor stem cells prior to bone marrow transplants can stave off toxicity when targeting acute myeloid leukemia with immunotherapy.