Gilotrif Approved for Metastatic EGFR-Positive NSCLC

The U.S. Food and Drug Administration (FDA) has approved Gilotrif™ (afatinib) for the treatment of metastatic non-small cell lung tumors that express specific types of epidermal growth factor receptor (EGFR) gene mutations.

Lung cancer remains the leading cause of cancer death in the United States. Non–small cell lung cancer (NSCLC) accounts for approximately 85% of all lung cancers. One sub-type of NSCLC is a type of lung cancer that is driven by a mutation in the epidermal growth factor receptor (EGFR) pathway. Each year in the United States over 18,000 individuals are diagnosed with NSCLC that harbors EGFR mutations. This type of cancer is associated with non-smokers and individuals of Asian descent.

EGFR is a protein located on the surface of many cancer cells that is involved in cancer growth. By blocking the EGFR pathway, targeted therapies help inhibit cancer growth. Gilotrif is a targeted therapy that blocks the EGFR pathway as well as the ErbB family of receptors that are associated with the EGFR pathway, including HER2 (ErbB2) and HER4 (ErbB4). Thus far, Gilotrif appears to block the EGFR pathway more thoroughly than other targeted therapies. It is intended for patients whose tumors express the EGFR exon 19 deletions or exon 21 L858R substitution gene mutations.

The approval was based on the results of a study that included 345 patients with advanced NSCLC who had EGFR mutations. Patients were randomly assigned to receive Gilotrif or standard combination chemotherapy treatment (pemetrexed and cisplatin). After a median follow-up of 8 months, Gilotrif delayed disease progression by more than 4 months over standard therapy—progression-free survival (PFS) was 11.1 months with Gilotrif, compared to 6.9 months with standard therapy.

Gilotrif was particularly beneficial to the 308 patients who had one of two common types of EGFR mutations (deletion 19 or L858R) that account of approximately 90 percent of all EGFR mutations. These patients experienced almost double the PFS—13.6 months in the Gilotrif group and 6.9 months in the standard chemotherapy group.

Patients receiving Gilotrif experienced a better quality of life than those receiving standard chemotherapy—and were slower to experience worsening of lung cancer symptoms such as cough and shortness of breath.

Common side effects of Gilotrif include diarrhea, skin breakouts that resemble acne, dry skin, itching (pruritus), inflammation of the mouth, skin infection around the nails (paronychia), decreased appetite, decreased weight, inflammation of the bladder (cystitis), nose bleed, runny nose, fever, eye inflammation and low potassium levels in the blood (hypokalemia). Serious side effects include diarrhea that can result in kidney failure and severe dehydration, severe rash,lung inflammation and liver toxicity.

Gilotrif was approved as part of the FDA’s priority review program, which allows an expedited six-month review of drugs that may offer major advances in treatment.

Gilotrif is being approved concurrently with the therascreen EGFR RGQ PCR Kit, a companion diagnostic that helps determine if a patient’s lung cancer cells express the EGFR mutations.


FDA approves new treatment for a type of late-stage lung cancer. FDA News Release]. U.S. Food and Drug Administration website. Available at:

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