Recent FDA Approval Is a Positive Signal for Personalized Medicine

6 Jul

By Michael Quigley, VP of Investor Research, LSN


Due to a recent FDA decision, immuno-oncology has proven to be a key area for personalized medicine approaches.  In this article, Next Phase will explore what this could mean for new technology development.

Many currently marketed cancer immunotherapies struggle due to high toxicity and response patterns that differ when compared to traditional therapies (1). Both in a research and clinical setting, identifying those patients most likely to respond to these and other new therapeutic approaches represents a massive opportunity for pharma companies.  This has given rise to increasing interest and investment in the field of companion diagnostics.

In May, the FDA approved Keytruda as the first immunotherapy cancer drug based on tumor specific molecular characteristics. While there have already been such approvals for genetic based markers, the FDA’s approval of Keytruda based on tumor specific characteristics offers shows the regulatory environment’s positive sentiment on other personalized approaches as well.  This is the first of such successes of a long developed shift away from the more traditional organ based approvals/research for cancer therapeutics.

While this shows obvious promise for large pharma companies developing these types of therapeutics it also shines a very positive light on companies looking at innovative ways of profiling tumor and patient subtypes.  These include including sequence-based diagnostics, multiple gene and protein signatures, multiplexed and digital PCR, microfluidic systems and novel signal detection approaches among others. These methods for identifying predictive biomarkers and patient stratification stand to move the field of immunotherapy forward, away from its current toxicity and efficacy issues and into an era of more personalized medicine.

Looking farther into the future implications of this approval is another positive sign into the early research current being done into what is known as “theranostics”. Where companion diagnostics are generally developed for an existing therapeutic agent the development of theranostics is a process involving the co-creation of therapeutics and a diagnostic to identify which patients will respond most positively. This is the next frontier for personalized medicine, where therapeutics are designed inherently to be effective in sub-populations of a disease or condition. Though this approach would potentially shrink the potential market for a new drug rather than aiming for “blockbuster” status, it also presents the opportunity to dramatically improve research and clinical trial efficiency for these compounds.

Overall this latest Keytruda approval provides a signal that the FDA is on board with this trend towards personalized medicine that researchers and drug developers have been moving towards for years.  In an industry that is so filled with risk and uncertainty, any form of reassurance from the regulatory environment is always welcome, and investors may see this as a positive signal for the sector.


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