Biomarker & CDx
September 13, 2021

Major FDA Approvals in Precision Oncology

Biomarker & CDx
August 5, 2020

Major FDA Approvals in Precision Oncology


Let's look at some of the recent FDA approvals like Merck’s blockbuster drug Keytruda, Genentech's cancer combo drug Phesgo, and some other drugs that have had a major impact on the precision oncology field.

Precision oncology is an innovative approach in cancer treatment based on the growing understanding of how cancers develop using molecular profiling. This method plays an essential part in discovering how genomes and other biomarkers are involved in the cause or development of cancer. This knowledge permits researchers to focus on these markers with the creation of precisely targeted therapies in oncology. There have been some interesting developments with recent approvals from the FDA we will take a look at here.

Keytruda Expansions

June 2020 saw the FDA expand the use of Merck’s (MSD) blockbuster drug Keytruda (pembrolizumab) as a first-line monotherapy in Microsatellite Instability-High or mismatch repair deficient (dMMR) metastatic colorectal cancer (mCRC).

In 2018, there were over 1.8 million new cases of colorectal cancer and around 5% of patients with mCRC have MSI-H or dMMR tumors. The disease remains incurable and most patients succumb to it. Identifying new indications for current drugs like Keytruda has the massive benefit of creating new therapeutic options without having to use the required expense, resources, and time nearly always involved in bringing a new drug to market. Keytruda was in fact the first cancer drug licensed by the FDA for targeting biomarkers in 2017 regardless of tumour type and earlier in June the FDA also gave a second approval for Keytruda based on another biomarker called tumour mutational burden load.

Results from the Keynote-177 trial showed that those receiving Keytruda lived twice as long without disease progression compared to those receiving standard-of-care chemotherapy or chemotherapy alongside Roche’s Avastin (bevacizumab) or Merck KgaA’s Erbitux (cetixumab). Competition in the mCRC market comes from Bristol Myers Squibb PD-1 inhibitor Opdivo (nivolumab). Total sales for Opdivo reached $6.7bn and $7.2bn in 2018 and 2019, whereas Keytruda hit $7bn and a whopping $11.1bn in the same time period.

Furthermore, Keytruda gained an additional 17 approvals or expansions from the FDA since the end of 2017, compared to Opdivo’s seven extra FDA approvals, giving Merck’s multi-purpose ‘umbrella’ drug a clear edge.


Genentech won FDA approval in late June this year for its injectable HER2-positive breast cancer combo drug, Phesgo. The treatment contains fixed doses of Perjeta (pertuzumab) and Herceptin (trastuzumab) and hyaluronidase, given alongside IV chemotherapy. The triple-combo drug can be injected in a patients home in just 8 minutes with 5 minute maintenance doses. This compares with the intravenous route taking 150 minutes and another 60-150 minutes for maintenance doses. The study found that a massive 85% of patients preferred the injections over IV.

The global breast cancer market is expected to grow to $38.4bn by 2025 and 2018 saw over 2 million new cases of breast cancer worldwide. Currently, new investigatory methods of treating the condition involve the use of finding and targeting novel and emerging breast cancer biomarkers.

Other Approvals

On June 18th the FDA also granted accelerated approval to Epizyme’s Tazverik (tazemetostat) for relapsed follicular lymphoma patients with an EZH2 mutation. Follicular lymphoma is a slow-growing blood cancer that affects the body’s white blood cells or lymphocytes. In trials patients with the EZH2 mutation saw an overall response rate of 69% to Tazverik, including 12% who achieved a complete response.

Epizyme CMO, Shefali Agarwal, commented at the time: “There remains no clear standard-of-care in relapsed and/or refractory follicular lymphoma as not all patients benefit from today’s available therapies.”

Finally, on June 16th Wyeth’s Mylotarg (gemtuzumab ozogamicin) received a new indication in CD33 positive acute myeloid leukemia. Wyeth is a subsidiary of pharma giant Pfizer, manufacturer of blockbusters Ibrance for breast cancer and cholesterol drug Lipitor. The study which involved 1,000 AML patients, showed results of an 8% increase in event free survival for Mylotarg versus chemotherapy. Gemtuzumab works by locking on to the CD33 protein and releases ozogamicin into the leukemia cells to damage and kill them. It is estimated there will be 60,000 new cases of leukemia in the U.S in 2020. of which nearly 20,000 will be classified as AML.

The new indication for Mylotarg means that drug can now be used in pediatric patients aged 1 month and older.


These recent approvals have had a major impact on the precision oncology field and our team continues to monitor developments in this space. Leaders in translational medicine, biomarkers and companion diagnostics can access comprehensive coverage on these and other drugs via the Larvol Veri platform to monitor biomarkers relevant to their team.


Larvol is a SaaS company that has been supporting the drug development and research teams in pharma and life sciences since 2004. Our newest decision and research-support tool, Larvol Veri,  is an oncology database with 1,000+ biomarkers, 200+ clinical diagnostic tests, and 50+ of the top diagnostic companies. Track your biomarkers at or sign-up for a free premium commercial trial here.

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