What Long-Term Trial Results Mean for ALK+ and EGFR+ Lung Cancer

Diagnosis and treatment are improving for people with non-small cell lung cancer (NSCLC). Targeted therapies are at the forefront of this progress. They are a form of “personalized” medicine.^1,2

Targeted therapy starts with a complete diagnostic assessment. This includes any genetic changes (mutations) in the cancer cells. Medicines then address the problems caused by your specific mutations. This approach is increasing survival rates by several years.^1,2

Long-term results from 2 major clinical trials offer even more hope to people with NSCLC. The results show dramatic improvements in how long people live without their cancer worsening. These treatments are changing the course of NSCLC.^3,4,10

New clinical trial data on targeted therapies

Studies have shown that 2 targeted therapies help people with certain types of NSCLC live longer without their cancer worsening.^3,4 Targeted therapies work by blocking specific proteins that help tumors grow. They only work if you have the genetic changes that the treatment targets.^1,2

The 2 targeted therapies are:^3-6

• Lorbrena® (lorlatinib): an anaplastic lymphoma kinase (ALK) inhibitor
• Tagrisso® (osimertinib): an epidermal growth factor receptor (EGFR) inhibitor

The US Food and Drug Administration (FDA) has already approved these drugs to treat certain people with NSCLC. New studies have been testing the long-term benefits of these drugs. This includes their effects on:^3,4

• Progression-free survival (PFS): how long someone lives without disease worsening
• Brain metastases: cancer cells spreading to the brain, which is a key challenge in NSCLC

Long-term benefits of Lorbrena in ALK-positive NSCLC

Lorbrena was first approved in 2018. In 2021, the FDA expanded its approval so it can be used as a first treatment for people with metastatic ALK-positive NSCLC. Metastatic cancer has spread from the place where it first grew. ALK-positive means the cancer cells have changes in the ALK gene.^6,7

Initial clinical trials compared Lorbrena with an older ALK inhibitor, Xalkori® (crizotinib). People taking Lorbrena went longer without their cancer worsening than people taking Xalkori. A follow-up 3 years later showed this benefit continued. After 3 years, about 64 percent of people treated with Lorbrena had not had disease progression, compared with 19 percent of people taking Xalkori.⁸

The latest results, shared in 2026, looked at 7 years of follow-up. They continue to show strong benefits, including:¹⁰

• A 7-year PFS rate of 55 percent, compared with 3 percent for Xalkori
• An 81 percent lower risk of disease progression or death
• A 94 percent lower risk of cancer growing in the brain

No new safety concerns emerged over 7 years. Overall, results show remarkable progress in treatment for ALK-positive NSCLC. Researchers are still following people in this study, including how long they live overall.¹⁰ Lorbrena can cause side effects, including high cholesterol and changes in mood, thinking, or speech. Talk with your doctor about what to expect.⁶

Benefits of Tagrisso for more people with EGFR-positive NSCLC

Tagrisso was first approved in 2015 and is now approved for several groups of people with EGFR-positive NSCLC, including people with metastatic disease. This means the cancer cells have an EGFR mutation (EGFR-positive). Tagrisso may be used with chemotherapy in this context. It may also be given after surgical removal of the tumor.^3,5,9

A clinical trial tested Tagrisso in people with unresectable stage III EGFR-positive NSCLC. Unresectable means that surgery cannot remove the cancer. Stage III means the cancer has also spread to nearby parts of the body.³

About 25 percent of people with NSCLC have stage III disease at diagnosis. Up to 90 percent of these people have unresectable disease. And about 1 in 3 people with unresectable stage III NSCLC have EGFR mutations. No targeted therapies were approved for this population when this study began.³

The clinical trial showed significant benefits of Tagrisso in this population. Results included:³

• Half of people taking Tagrisso lived more than 3 years without their cancer worsening, compared with less than 6 months with placebo
• An 84 percent lower risk of disease progression or death
• An 8 percent risk of brain metastases, compared with 29 percent with placebo

This study showed no new safety concerns with Tagrisso. Overall, the results show meaningful benefits. This is important for a population that may not respond well to chemotherapy. Based on these results, the FDA approved Tagrisso in September 2024 for people with unresectable stage III EGFR-positive NSCLC whose cancer has not gotten worse after chemoradiation. This study is ongoing, and more data will be reported in the future.^3,11 Tagrisso can cause side effects, including diarrhea, rash, dry skin, and nail changes. Less often, it can cause serious lung or heart problems. Talk with your doctor about what to expect.⁵