Array BioPharma has achieved a significant regulatory milestone as its innovative combination therapy for metastatic colorectal cancer (mCRC) has been granted breakthrough therapy designation by the U.S. Food and Drug Administration (FDA). The designation specifically targets the treatment regimen combining encorafenib (BRAFTOVI), binimetinib (MEKTOVI), and cetuximab for mCRC patients with the BRAFV600E mutation, following the failure of one to two prior lines of therapy.

This breakthrough therapy designation is particularly significant for patients with mCRC who test positive for the BRAFV600E mutation—a group facing a mortality risk more than twice that of mCRC patients without the mutation. Currently, there are no treatments specifically approved for this subset of mCRC patients, marking a substantial unmet medical need within the oncology community.

The FDA’s breakthrough therapy designation is intended to expedite the development and review of drugs which may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. The designation for Array BioPharma’s combination therapy is based on encouraging Phase 3 BEACON CRC safety lead-in data, which indicated promising efficacy and safety profiles.

Victor Sandor, Chief Medical Officer of Array BioPharma, expressed his optimism: “We are delighted that the FDA has recognized the potential of this combination for patients with BRAFV600E-mutant metastatic colorectal cancer. As there are no regimens approved specifically for BRAFV600E-mutant mCRC, this designation provides us with the opportunity to work closely with the FDA to potentially accelerate our effort to bring an important treatment option to these patients in critical need.”

The breakthrough designation was supported by data from the BEACON CRC trial, where the median overall survival (OS) had not yet been reached at a follow-up of 12.6 months. The one-year overall survival rate for the cohort was 62%, with a median progression-free survival (mPFS) of 8 months. Notably, the confirmed overall response rate (ORR) was 48%, and among patients who had only one prior line of therapy, the ORR rose to 62%.

The combination therapy was generally well-tolerated with no unexpected toxicities, reinforcing its potential as a viable treatment option for this challenging cancer subtype. As the trial continues, the oncology community watches closely, hopeful for a new standard of care that could significantly extend life and improve quality of life for patients with BRAFV600E-mutant mCRC.

The ongoing development and regulatory review of this treatment reflects Array BioPharma’s commitment to addressing gaps in cancer care through innovative targeted therapies.

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