The Personalized Medicine Coalition has released its annual report on precision medicine trends at the Food and Drug Administration (FDA). Their analysis covers FDA approvals in 2019, including new milestones in the continued pursuit of the precision medicine paradigm—the right drug for the patient at the right time, and at the right dose. While these findings illustrate a significant dip in novel precision medicine approvals from 2018 (down from 42% to 25%), we’re still seeing remarkable innovation in areas like siRNA and cystic fibrosis.
Here are 8 key takeaways from the report:
- 12 total personalized medicines were approved by FDA in 2019, including 11 new molecular entities (NMEs) and 1 new gene therapy
- Personalized medicines topped 20% of FDA approvals for the 6th year in a row. FDA CDER approved 11 NMEs in 2019, representing 25% of its 44 therapeutic approvals; FDA CBER approved 1 new gene therapy. Note for context that the percentage calculations only include year-over-year comparisons concerning CDER approvals.
- 5 of the 12 newly approved personalized medicines are designed to reverse previously unmitigated root causes of certain congenital diseases such as spinal muscular atrophy (SMA), Duchenne muscular dystrophy, cystic fibrosis, and sickle cell disease.
- FDA approved many significant new personalized medicine indications for previously existing drugs in 2019 for new molecularly defined subsets of patients. These approvals redefine the drugs’ intended populations and often provide patients with more effective personalized treatment options. If these new indications were added to the list of ‘new’ personalized medicines in 2019, the number would more than double, increasing by 15.
- 2019 marked the emergence of siRNA treatments with the approval of Givlaari (givosiran) for the second FDA approval of a new class of personalized medicine drugs called small interfering ribonucleic acid (siRNA) treatments, which selectively target and silence a portion of RNA involved in causing disease.
- Several biosimilars for personalized medicines were also approved in 2019, include biosimilars for Rituxan (rituximab) and Gleevec (imatinib).
- In 2019, FDA’s CDRH approved or cleared seven new or expanded in vitro diagnostic tests that underpin personalized medicine strategies.
- For the first time in 2019, FDA qualified a digital medical device development tool of a personalized medicine test type. The OsiriX CDE Software Module is the first biomarker test for brain injury and may help innovators more efficiently enroll patients, based on their individual characteristics, in clinical trials of therapeutic medical devices intended to be used to treat mild traumatic brain injury.
For more details, the coalition’s report can be accessed here.