FDA Set Record in 2017 for Targeted Therapy Approvals

For the he fourth consecutive year, targeted therapies accounted for more than 20 percent of all new drug approved by the U.S. Food and Drug Administration (FDA), according to the Personalized Medicine Coalition's (PMC's) recently released its 2017 Progress Report, Personalized Medicine at the FDA. In 2017, the FDA approved 16 new molecular entities—34 percent of all new drugs, agents, or therapeutic biologics approved in 2017.

"Even the large number of newly approved therapies classified as personalized medicines in 2017 does not provide the whole picture," writes PMC in the report. "The growing list of personalized medicines available to doctors and their patients also includes many significant new personalized medicine indications for [15] previously approved drugs in 2017."

PMC specifically highlights the importance of the expanded approval of Keytruda (pembrolizumab) for all solid tumor types in advanced cancers with microsatellite instability-high or mismatch repair deficiency (is particularly significant as it marks the first time an oncology drug has been approved based on a biomarker, regardless of where the tumor is located in the body.

As DTET has previously reported, PMC also highlights other "significant" FDA approvals, including the first:

• Authorization for marketing of health-related genetic tests directly to consumers—23andMe's Personal Genome Service Genetic Health Risk tests.

• Approval of personalized medicine biosimilar for Herceptin (trastuzumab), which was first approved for HER2-positive breast cancer in 1998.

• Joint approval and coverage decision with the FDA and the Centers for Medicare and Medicaid Services for Foundation Medicine's FoundationOne CDx test, which uses next-generation sequencing technology.

"While ongoing challenges in the areas of scientific discovery, diagnostic regulatory policy, coverage and reimbursement, and implementation of new technologies into clinical practice are still outstanding," PMC writes, "the science leading health care away from one-size-fits-all, trial-and-error medicine and toward the utilization of molecular information to improve outcomes and make the U.S. health system more efficient is clear."

Takeaway: The FDA's record number of personalized medicine-related approvals in 2017 shows the commitment by the pharmaceutical and diagnostic industries, as well as the FDA, towards advancing targeted treatments.