FDA's Personalized Medicine Approvals on the Rise

According to the recently released Personalized Medicines Coalition (PMC) 2017 Progress report, the FDA approved a record number of 16 new personalized medicines in 2017.

In fact, PMC reported that in the past 4 years, more than 1 in every 4 drug approvals has been of a personalized medicine. In 2017, the agency also approved the first personalized medicine biosimilar in the United States: trastuzumab-dkst, (Ogivri) received FDA approval in December 2017 to treat HER2-positive breast cancer. Trastuzumab-dkst is referenced on trastuzumab (Herceptin), which received FDA approval in 1998.

In addition, the agency approved the first 3 gene therapies for the first time in the United States. Two of the gene therapies, tisagenlecleucel (Kymriah) and axicabtagene ciloleucel (Yescarta), are CAR T-cell treatments for acute lymphoblastic leukemia and large B-cell lymphoma, respectively. The final gene therapy, voretigene neparvovec-rzyl (Luxturna) was approved for the treatment of retinol dystrophy.

“We’re increasingly able to identify patient benefit earlier in the development process because of the ability to better target medicines to the underlying mechanisms of disease,” said FDA Commissioner Scott Gottlieb, MD, of these approvals.

The FDA also expanded indications in 2017 for 15 different personalized medicines, including pembrolizumab (Keytruda). Pembrolizumab is now indicated to treat all solid tumor types in advanced cancers with microsatellite instability-high or mismatch-repair deficiency. The PMC report notes that this marks the first time an oncology drug has been approved based on a biomarker, regardless of where the tumor is located within the body.

Notably, for the second time in their histories, the FDA and CMS announced a joint approval and coverage decision under the Parallel Review program in 2017. This approval was granted to a next-generation sequencing (NGS) testing platform, Foundation Medicine’s FoundationOne CDx. Additionally, for the first time, the FDA authorized the commercialization of health-related genetic tests that are sold directly to consumers, such as the 23andMe Personal Genome Service Genetic Health Risk test.

“FDA has an important role to play in advancing this targeted approach to treating disease by building a modern framework that ensures we’re providing the guidance and resources needed to efficiently develop these novel products using new technology,” said Gottlieb.