The US Food and Drug Administration (FDA) has granted orphan drug designation to lurbinectedin, a novel anticancer agent, for the treatment of patients with small-cell lung cancer (SCLC), according to a press release.1

Recent data from a phase 2 basket study ( Identifier: NCT02454972) presented during a poster session at the 2018 American Society of Clinical Oncology Annual Meeting in Chicago, Illinois, suggested that lurbinectedin may be a safe and effective second-line option for patients with SCLC.

Researchers administered lurbinectedin as a single agent to 61 patients with recurrent SCLC. The study group had an objective response rate of 39.3%, a median duration of response of 6.2 months, and a median overall survival of 12 months. The most frequently observed adverse event was myelosuppression, with 39% and 9% of patients experiencing grade 3 to 4 neutropenia and febrile neutropenia, respectively.2

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Lurbinectedin is a selective inhibitor of RNA polymerase II, a critical enzyme in the transcription process that is over-activated in tumors with transcription addiction, and leads to DNA double-strand breaks and apoptosis. It is currently undergoing clinical investigation.

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SCLC is a highly aggressive disease that is diagnosed in 34,000 patients and accounts for nearly 18% of all lung cancer cases in the United States every year. The FDA grants orphan drug designation to therapies that may be safe and effective in the treatment of diseases that affect less than 200,000 patients per year in the United States.


  1. The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PharmaMar’s lurbinectedin [press release]. Madrid, Spain: PR Newswire; August 3, 2018. Accessed August 3, 2018.
  2. PharmaMar presents new results with lurbinectedin as a single agent in patients with recurrent small-cell lung cancer [press release]. Madrid, Spain: PharmaMar; June 4, 2018. Accessed August 3, 2018.