US FDA approves TAGRISSO® (osimertinib) blood-based T790M companion diagnostic test

AstraZeneca today announced that the US Food and Drug Administration (FDA) has approved a blood-based companion diagnostic for TAGRISSO® (osimertinib). The companion diagnostic for TAGRISSO is the only FDA-approved and clinically validated companion diagnostic test that uses either tissue or a blood sample to confirm the presence of a T790M mutation in patients with metastatic epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC), who have progressed on or after an EGFR tyrosine kinase inhibitor (TKI) medicine.

The approval provides a new, non-invasive option to identify patients with metastatic EGFR T790M mutation-positive NSCLC, ensuring that those patients who may not be suitable for biopsy procedures have an opportunity to be tested. Blood-based testing for the presence of the mutation is recommended only when a tumor biopsy cannot be obtained. Patients who test negative for the T790M mutation with the blood-based test, and their physicians, should re-evaluate the feasibility of tissue-based testing to confirm the presence of the EGFR T790M mutation.

The companion diagnostic, cobas® EGFR Mutation Test v2, was developed by Roche Molecular Systems. The test enables identification of patients who have the T790M mutation at disease progression, and is initially available through Baystate Health, Carolinas HealthCare System, Laboratory Corporation of America® Holdings (LabCorp®), and PhenoPath.

“Blood-based testing has the potential to rapidly identify patients eligible for targeted therapy, who may not be eligible for biopsy. Availability of this blood-based test may help aid treatment decisions,” said Balazs Halmos, MD, Montefiore Medical Park, Albert Einstein College of Medicine.

“The availability of an FDA-approved, blood-based companion diagnostic is a tremendous step forward for patients with lung cancer in need of a high-quality test that provides results with a rapid turnaround time. This development offers an important option for the identification of the T790M mutation in patients with metastatic EGFR mutation-positive NSCLC who have progressed on an EGFR TKI medicine, for whom a tissue biopsy may not be feasible,” said Andrew Coop, Vice President, US Medical Affairs, Oncology, AstraZeneca. “Delivering targeted therapies, such as TAGRISSO, to the right patients at the right time demonstrates our commitment to testing and quality companion diagnostics.”

Nearly two-thirds of cases of progression with first-generation EGFR TKIs are related to an acquired EGFR T790M mutation — for which there have been limited treatment options in the past. TAGRISSO is the only FDA-approved targeted medicine for the treatment of patients with metastatic EGFR T790M mutation-positive NSCLC who have progressed on or after an EGFR TKI medicine, and was approved by the FDA in November 2015. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

The TAGRISSO tolerability profile showed that no individual severe grade 3+ adverse events occurred at ≥ 3.5%. The most common adverse events were generally mild to moderate and included diarrhea (42% all grades; 1.0% grade 3/4), rash (41% all grades; 0.5% grade 3/4), dry skin (31% all grades; 0% grade 3/4), and nail toxicity (25% all grades; 0% grade 3/4).

Important Safety Information About TAGRISSO® (osimertinib)

  • There are no contraindications for TAGRISSO.
  • Interstitial Lung Disease (ILD)/Pneumonitis occurred in 3.3% and were fatal in 0.5% of 813 TAGRISSO patients. Withhold TAGRISSO and promptly investigate for ILD in any patient presenting with worsening of respiratory symptoms indicative of ILD (e.g., dyspnea, cough and fever). Permanently discontinue TAGRISSO if ILD is confirmed
  • QTc interval prolongation occurred in TAGRISSO patients. Of the 411 patients in two Phase II studies, 0.2% were found to have a QTc greater than 500 msec, and 2.7% had an increase from baseline QTc greater than 60 msec. Conduct periodic monitoring with ECGs and electrolytes in patients with congenital long QTc syndrome, congestive heart failure, electrolyte abnormalities, or those who are taking medications known to prolong the QTc interval. Permanently discontinue TAGRISSO in patients who develop QTc interval prolongation with signs/symptoms of life threatening arrhythmia
  • Cardiomyopathy occurred in 1.4% and were fatal in 0.2% of 813 TAGRISSO patients. Left Ventricular Ejection Fraction (LVEF) decline >10% and a drop to <50% occurred in 2.4% of (9/375) TAGRISSO patients. Assess LVEF before initiation and then at 3 month intervals of treatment. Withhold TAGRISSO if ejection fraction decreases by 10% from pretreatment values and is less than 50%. For symptomatic congestive heart failure or persistent asymptomatic LV dysfunction that does not resolve within 4 weeks, permanently discontinue TAGRISSO.
  • Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during TAGRISSO treatment and for 6 weeks after the final dose. Advise males with female partners of reproductive potential to use effective contraception for 4 months after the final dose.
  • The most common adverse reactions (>20%) observed in TAGRISSO patients were diarrhea (42%), rash (41%), dry skin (31%) and nail toxicity (25%).

TAGRISSO is indicated for the treatment of patients with metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive non-small cell lung cancer (NSCLC), as detected by an FDA-approved test, who have progressed on or after EGFR tyrosine kinase inhibitor therapy.

This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Please see accompanying complete Prescribing Information including Patient Information.


About T790M Blood-Based Testing
Overall detection rates for the EGFR T790M mutation from plasma and tissue samples are broadly concordant; differences in test results can arise from biological (tumor heterogeneity and differential shedding) and technical (assay methodology) factors. A blood sample that tests negative for T790M mutation does not necessarily mean a patient is ineligible for TAGRISSO, as a positive result may still be obtained with tissue, if the patient is eligible for biopsy. Blood-based testing for T790M has been conducted in multiple studies, including in a cross-platform comparison of leading technologies to support the clinical development of TAGRISSO (Phase I/II AURA trials).  

About Non-Small Cell Lung Cancer
Lung cancer is the leading cause of cancer death among both men and women, accounting for about one-third of all cancer deaths — more than breast, prostate and colorectal cancers combined. Lung cancer has a five-year survival rate that is less than 20 percent. Approximately 85 percent of all lung cancers in the US are NSCLC; 10 to 15 percent of these are EGFR mutation-positive. Approximately two-thirds of patients treated with an EGFR TKI medicine will acquire resistance related to the T790M mutation.

TAGRISSO® (osimertinib) 80mg once-daily tablet is the first medicine indicated for the treatment of metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive NSCLC, as detected by an FDA-approved test, who have progressed on or after EGFR TKI therapy.

About the TAGRISSO Development Program
TAGRISSO® (osimertinib) is being studied in the confirmatory trial, AURA3, an open label, randomized Phase III study designed to assess the efficacy and safety of TAGRISSO versus platinum-based doublet chemotherapy in patients with EGFR T790M mutation-positive, locally advanced or metastatic NSCLC who have progressed following prior therapy with an EGFR TKI. TAGRISSO is also being investigated in the adjuvant setting and in the metastatic first-line setting, including in patients with brain metastases, as well as in combination with other compounds.

About AstraZeneca in Oncology
AstraZeneca has a deep-rooted heritage in oncology and offers a quickly growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With at least six new medicines to be launched between 2014 and 2020 and a broad pipeline of small molecules and biologics in development, we are committed to advancing New Oncology as one of AstraZeneca’s six Growth Platforms, focused on lung, ovarian, breast and blood cancers. In addition to our core capabilities, we actively pursue innovative partnerships and investments that accelerate the delivery of our strategy, as illustrated by our investment in Acerta Pharma in hematology.

By harnessing the power of four scientific platforms — immuno-oncology, the genetic drivers of cancer and resistance, DNA damage repair and antibody drug conjugates — and by championing the development of personalized combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.

About AstraZeneca
AstraZeneca is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialization of prescription medicines, primarily for the treatment of diseases in three therapy areas – Respiratory and Autoimmunity, Cardiovascular and Metabolic Diseases, and Oncology. The company is also active in inflammation, infection and neuroscience through numerous collaborations. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. For more information please visit:

About Roche
Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people’s lives.

Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalized healthcare – a strategy that aims to fit the right treatment to each patient in the best way possible.

Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. Twenty-nine medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Roche has been recognized as the Group Leader in sustainability within the Pharmaceuticals, Biotechnology & Life Sciences Industry seven years in a row by the Dow Jones Sustainability Indices.

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2015 employed more than 91,700 people worldwide. In 2015, Roche invested CHF 9.3 billion in R&D and posted sales of CHF 48.1 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit


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3245603 Last Updated 9/16