The FDA has approved the use of Gilotrif (afatinib) to treat patients with non-small cell lung cancer (NSCLC) who have certain epidermal growth factor receptor (EGFR) gene mutations in their tumors.

Lung cancer is one of the leading cancer-killers, with an estimated 159,480 people dying from the disease this year in the U.S. alone. Most cases of lung cancer are NSCLC (around 85 percent) and nearly one in 10 who have NSCLC have a EGFR gene mutation - most of them expressing exon 21 L858R substitution or EGFR exon 19 deletions.

As a a tyrosine kinase inhibitor, Gilotrif works by blocking proteins that are necessary for cancer cells to develop. The drug will be approved exclusively for patients with tumors that express the EGFR exon 21 L858R substitution or exon 19 deletions or gene mutations.

The drug was reviewed under the FDA's priority review program which speeds up the review for medications that may provide effective therapy when no existing treatment option exists.

The therascreen EGFR RGQ PCR Kit is used to determine whether a person's tumor cells express an EGFR mutation.

Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research, said that "today's approvals further illustrate how a greater understanding of the underlying molecular pathways of a disease can lead to the development of targeted treatments. Gilotrif is the second drug approved this year for patients with untreated metastatic NSCLC whose tumors have the EGFR exon 19 deletions or exon 21 L858R substitution mutations."

Alberto Gutierrez, Ph.D., director of the Office of In Vitro Diagnostics and Radiological Health in the FDA's Center for Devices and Radiological Health commented that "the approval of companion diagnostic tests and drugs are important developments in oncology, as they help us bring safe and effective treatments to patients who need them."

Tumor samples from participants in the clinical trial were used to assess the efficacy of the "therascreen EGFR RGQ PCR Kit" in detecting EGFR mutations.

Earlier this year, the FDA approved the cobas EGFR Mutation Test that also detects epidermal growth factor receptor (EGFR) gene mutations. The cobas test is a companion diagnostic for the cancer drug Tarceva (erlotinib).

A total of 345 people with NSCLC whose tumors had EGFR mutations participated in the study. Those who took Gilotrif experienced a 4.2 month delay in tumor growth compared to those who received chemotherapy.

Side effects of Gilotrif include:

  • Fever
  • Severe rash
  • Decreased appetite
  • Eye inflammation
  • Decreased weight
  • Lung inflammation
  • Decreased weight
  • Nose bleed
  • Skin infection

Boehringer Ingelheim Pharmaceuticals, Inc is marketing the drug Gilotrif.

Kevin Lokay, vice-president and business unit head, Oncology, Boehringer Ingelheim Pharmaceuticals, Inc., said:

"We are truly excited to be able to offer GILOTRIF as a new treatment option for these patients. This approval is an achievement for Boehringer Ingelheim Oncology and the many teams and individuals who committed themselves to developing this therapy based on its potential identified in the clinical trial program. GILOTRIF marks the first, of what we expect will be many, oncology products to emerge from our research and development program."