Project name: afatinib
Disease: Non-small cell lung cancer
Peak sales estimate: Not available
Approved: July 12
Company: Boehringer Ingelheim
Boehringer Ingelheim's afatinib tablets, marketed as Gilotrif, gained an FDA blessing in July after the agency evaluated the drug under its priority review program, which provides a fast-track route for promising drugs to treat conditions that lack adequate available treatments.
The approval was a strategic win for Boehringer, which has a number of late-stage cancer drugs in its pipeline, indicating a foray into the personalized cancer treatment sector. In September, the German drugmaker caught the FDA's eye again, earning a breakthrough therapy designation for volasertib in elderly patients with previously untreated acute myeloid leukemia.
"It marks the first of what we hope will be many products to emerge from our oncology research and development program, and underscores our continued commitment to translating innovative science into new treatment options for patients," Klaus Dugi, Boehringer's corporate senior vice president of medicine, said in a July statement.
One of the more promising candidates to come out of the 2012 ASCO annual conference, afatinib is approved to treat patients with late-stage, or metastatic, non-small cell lung cancer (NSCLC) whose tumors express specific types of epidermal growth factor receptor (EGFR) gene mutations.
To determine whether patients have these types of mutations, the FDA also approved a Qiagen ($QGEN) diagnostic called a therascreen alongside afatinib that identifies patients whose lung cancer cells express EGFR.
Afatinib, an ErbB family blocker, is a tyrosine kinase inhibitor designed to block proteins that spur cancer growth. The drug works by irreversibly inhibiting EGFR, as well as other mutations in the ErbB family that are known to play a critical role in the growth and spread of highly deadly cancers like NSCLC. It's most likely to work in patients whose tumors express EGFR exon 19 deletions or exon 21 L858R substitution gene mutations, as detected by the Qiagen test.
Lung cancer is the leading cause of cancer-related death among men and women, and NSCLC is the most common type, making up about 85% of all lung cancers. About 10% of NSCLC cases have EGFR gene mutations present.
Non-small cell lung cancer has proven to be a tough target for drug developers to crack, and the rate of survival has remained poor. Historically, late-stage metastatic NSCLC has been treated with chemotherapy, but new targeted therapies coming on the market for patients with specific biomarkers are starting to chip away at the larger patient population, providing more tailored treatment plans to smaller, niche groups.
In a clinical study of 345 participants with metastatic NSCLC whose tumors harbored EGFR mutations, trial participants who took afatinib as a first-line treatment compared to standard chemotherapy drugs lived for 11.1 months versus 6.9 months for those treated with pemetrexed and cisplatin. Patients with tumors harboring the two most common EGFR mutations--Del19 or L858R--taking afatinib lived for 13.6 months versus 6.9 months for those in the control arm.
Gilotrif was the second drug approved in 2013 for patients with untreated metastatic NSCLC whose tumors have the EGFR exon 19 deletions or exon 21 L858R substitution mutations. The first was Astellas' Tarceva (erlotinib), which secured an expanded approval in May as a first-line treatment.
Afatinib is currently in phase III development in head and neck cancer and breast cancer. -- Emily Mullin (email | Twitter)
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