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Novartis Nets Tumor-Agnostic Approval for Tafinlar-Mekinist Combo

NEW YORK – The US Food and Drug Administration on Wednesday granted accelerated approval to Novartis' Tafinlar (dabrafenib) plus Mekinist (trametinib) for advanced, refractory patients with any type of solid tumor as long as it harbors a BRAF V600E mutation.

With this approval, the BRAF/MEK-inhibitor combination is an option for adults and children over age 6 whose cancers are metastatic or unresectable and who have no satisfactory treatment options. Patients with colorectal cancer, of note, are excluded from the latest tissue-agnostic indication. The FDA-approved labeling for the drugs notes that the combination "is not indicated for treatment of patients with colorectal cancer because of known intrinsic resistance to BRAF inhibition."

This is the first time a BRAF/MEK inhibitor combination has garnered a tissue-agnostic approval. However, this is the sixth time the agency has approved a tissue-agnostic indication for refractory cancer patients as a last-ditch option. Other tissue-agnostic indications are Merck's Keytruda (pembrolizumab) for microsatellite instability high/mismatch repair-deficient tumors and tumors with high tumor mutation burden; GlaxoSmithKline's Jemperli (dostarlimab) for MSI-high/dMMR tumors; and Bayer's Vitrakvi (larotrectinib) and Genentech's Rozlytrek (entrectinib) for NTRK fusion-positive solid tumors.

The agency granted tissue-agnostic approval to Tafinlar-Mekinist based on data from the multi-cohort Phase II ROAR and NCI-MATCH trials. In these studies, around 80 percent of patients with BRAF V600E-mutated tumors responded to the combination, including those with both high- and low-grade glioma, biliary tract cancer, and certain gynecologic and gastrointestinal cancers. The FDA also considered data from a study, dubbed X2101, which showed the drug combination's benefit in pediatric patients.

The approval follows data presented at the American Society of Clinical Oncology's annual meeting earlier this month demonstrating Tafinlar-Mekinist's benefit in pediatric patients with low-grade gliomas.

"The combination of dabrafenib and trametinib demonstrated meaningful efficacy in multiple BRAF-positive tumor types, including in some patients with rare cancers who have no other treatment options available," Vivek Subbiah, medical director of MD Anderson Cancer Center's Clinical Center for Targeted Therapy, said in a statement. "Physicians should consider a BRAF test as a routine diagnostic step that could enable a new option for treating patients with many solid tumors."

The BRAF V600E mutation has been shown to drive tumors across 20 different cancer types. The targeted treatment combination is already approved for certain patients with BRAF V600 mutation-positive melanoma, non-small cell lung cancer, and thyroid cancer.