(Gerd Altmann, Pixabay)
31 January 2018. A report on Food and Drug Administration approvals of new therapies says the agency is accelerating its reviews of treatments designed to address the specific molecular composition of individuals. The advocacy group Personalized Medicine Coalition in Washington, D.C. issued its report yesterday on FDA approvals in 2017.
In 2017, FDA lists approval of 46 new molecular entities, the official name for treatments with a different mechanism for action or not derived from an earlier product, by the agency. Of the 46 approvals, says the Coalition’s report, 16 of the new therapies, or 35 percent, require the presence of a particular biomarker in the patient, thus qualifying them as precision medicines. More than half of the approved precision therapies, 9 of 16, are for cancer, while the other personalized medicines treat neurological disorders or rare diseases. In another 15 cases, FDA approved expanding the diseases treated with existing drugs, but first requiring specific biomarkers.
FDA also in 2017 approved 3 gene therapies, where a patient’s missing or defective genes are replaced with healthy genes transplanted into affected cells. Of the 3 gene therapies, 2 are for blood-related cancers — Kymriah by Novartis for B-cell acute lymphoblastic leukemia and Yescarta by Gilead Sciences for relapsed or refractory large B-cell lymphoma. Science & Enterprise covered development and testing of these gene therapies over the past few years, as well as the third gene therapy, Luxturna by Spark Therapeutics, approved by FDA in December 2017 to treat a specific form of retinal dystrophy.
In addition, says the report, FDA took actions other than new therapy approvals that support precision medicine. In 2017, FDA approved a biosimilar therapy, a substitute biologic treatment acting much like the original, in this case Herceptin to treat HER2-positive breast cancer, first approved in 1998. The agency also approved for the first time direct-to-consumer genetic tests offered by the commercial genomics provider 23andMe that report on genetic health risks for 10 disorders. FDA used that approval to refine its processes for reviewing tests for genetic health predispositions marketed to consumers.
A related action by FDA approved genomic tests for solid tumor cancers offered by Foundation Medicine, which in December 2017 was simultaneously recommended for payments by Medicare to cover 5 types of cancer in advanced stages. The report says it’s only the second time FDA and Centers for Medicare and Medicaid Services, both under the Department of Health and Human Services, coordinated their activities to make a joint announcement.
An item to watch in next year’s report is development of regulatory guidance by FDA for review of clinical trials for treatments and diagnostics related to precision medicine. As reported by Science & Enterprise in mid-December 2017, the agency issued draft guidance for public comment, indicating its early thinking on rules for clinical trials for new treatments that target genetic mutations rather than disease states. The agency also released a companion document for comment on diagnostic tests that determine a patient’s status for taking part in clinical studies of therapies based on genomic factors.
More from Science & Enterprise:
- FDA to Release More Accessible Clinical Trial Data
- CAR T-Cells Shown Effective in Long-Term Studies
- FDA Approves Precision Solid Tumor Cancer Diagnostics
- FDA Sets Genetic Health Test Review Process
- Guidelines Designed for CAR T-Cell Cancer Therapies
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