Personalized Medicine at FDA: The Scope & Significance of Progress in 2022 | Foley & Lardner LLP
The Personalized Medicine Coalition (PMC) recently released its annual report on the progress of precision medicine as measured by precision medicine FDA approvals in 2022 (“Report”). As summarized in the Report, 2022 milestones include progress in diagnostics, therapeutics and new treatment modalities.
1. 12 new drugs identified as personalized medicines were approved in 2022, which represented about 34 percent of all newly approved drugs last year.
2. 5 new gene or cell based therapies were approved in 2022. These include treatment of rare genetic disorders with few other treatment options – beta thalassemia, hemophilia B, and cerebral adrenoleukodystrophy, refractory multiple myeloma, and certain types of non-muscle invasive bladder cancer.
3. 12 diagnostic testing systems were cleared or approved. Of the 12, three were new blood based biomarker tests for personalized cancer therapies. In addition, several of the 2022 approved indications will advance tumor-agnostic testing to guide therapeutic interventions for certain cancer patients.
4. Tumors with KRAS mutations were once considered resistant to therapy. However, in 2022, a new therapy to treat non-small cell lung cancer harboring KRAS G12C genetic mutations was approved, now the second targeted therapy for these cancers.
5. FDA also released two draft guidance documents that will serve to advance the developing fields of gene therapy and chimeric antigen receptor therapy. The Report indicates that the “documents promise to further enhance the efficacy with which product developers can develop submissions for this groundbreaking class of personalized treatments.”
6. The first of a new class of drugs – T cell engagers – was approved. The drug treats a rare form of eye cancer termed HLA-A*01:01 positive uveal melanoma. T cell engagers bind to a protein found on the tumors of patients to elicit an immune response.
PMC’s report concludes that the continued development of personalized therapies continues to improve patient outcomes and make health care more efficient, but such progress must not be taken for granted, urging policymakers to continue to support policies that encourage advancement in the field.