Orphan designated precision drug approved in US for NSCLC

The US Food and Drug Administration (FDA) has approved orphan drug encorafenib (Braftovi) with binimetinib (Mektovi) for adult patients with metastatic non-small cell lung cancer (NSCLC) with a BRAF V600E mutation. The FDA also approved the FoundationOne CDx (tissue) and FoundationOne Liquid CDx (plasma) as companion diagnostics for encorafenib with binimetinib. This is the second therapy the FDA has approved for NSCLC in recent weeks. In September, the regulator granted fast track designation to Mythic Therapeutics’ investigational cMET-targeting antibody-drug conjugate, MYTX-011. Efficacy was evaluated in 98 patients with metastatic NSCLC with BRAF V600E mutation enrolled in PHAROS, an open-label, multicentre, single-arm study. Prior BRAF or MEK inhibitors was not allowed. Patients received encorafenib and binimetinib until disease progression or unacceptable toxicity. The major efficacy outcome measures were objective response rate (ORR) per RECIST v1.1 and duration of response (DoR), as assessed by an independent review committee. Among 59 treatment-naïve patients, ORR was 75%; median DoR was not estimable (NE). Among 39 previously treated patients, ORR was 46% with a median DoR of 16.7 months. Precision medicine for NSCLC “BRAF V600E mutations identify a unique subtype of metastatic non-small cell lung cancer that presents an actionable biomarker that precision medicines like Braftovi + Mektovi combination therapy can help address,” said Gregory Riely, Vice Chair, Clinical Research in the Department of Medicine at Memorial Sloan Kettering Cancer Center (MSK) and PHAROS investigator. “The PHAROS trial demonstrated that these patients could benefit from Braftovi + Mektovi targeted therapy regardless of their prior treatment history.” The drugs were developed by Array BioPharma, which has been a wholly owned subsidiary of Pfizer since 2019. Pfizer has exclusive rights to market the drugs in the US, Canada, and all countries in Latin America, Africa, and the Middle East. Ono Pharmaceutical has exclusive rights to commercialise both products in Japan and South Korea, while Medison has exclusive rights in Israel, and Pierre Fabre has all rights in all other countries, including Europe and Asia-Pacific.