A drug that treats tumors based on a genetic marker rather than which tissue they affect has won approval in Europe.
On Monday, the European Medicines Agency approved Leverkusen, Germany-based Bayer’s Vitrakvi (larotrectinib), for cancers that display a genomic abnormality known as an NTRK fusion. The drug was originally developed under a partnership between Bayer and Loxo Oncology, the latter of which Lilly acquired in January for $8 billion. The EMA regulates drugs across the European Union and also the European Economic Area countries of Norway, Iceland and Liechtenstein.
The drug received Food and Drug Administration approval last November in the U.S., where it was the second drug to win a tumor-agnostic label, after Merck & Co. won approval for Keytruda (pembrolizumab) in microsatellite instability-high/mismatch repair-deficiency tumors. However, it was the first drug to win a tumor-agnostic label as its sole indication. And last month, Roche’s Rozlytrek (entrectinib) became the third drug to win a biomarker-based label. Rozlytrek targets NTRK fusions as well as ROS1 in lung cancer and initially won approval from Japanese regulators, in June.
In clinical trials, Vitrakvi has shown a response rate of 72 percent – including a 16 percent complete response rate – among patients taking the drug, with three-quarters still on treatment after one year. The company plans to present updated durability data at Saturday at the European Society for Clinical Oncology’s annual meeting in Barcelona, Spain. Such a high response rate is due not only to the drug’s specificity, but to its targeting of NTRK, a well-known genetic driver of cancers. NTRK fusions are expressed across a wide variety of solid tumors, but usually in a very rare percentage of patients.
Earlier this month, Lilly presented data for another drug, LOXO-292 (selpercatinib), at the World Conference on Lung Cancer, which also took place in Barcelona. Selpercatinib targets RET fusions, another genetic driver of cancers, and the data presented showed an overall response rate of 68 percent among patients with non-small cell lung cancer. The company plans to submit a regulatory approval application for the drug by the end of this year. Another company, Blueprint Medicines, has a competing RET inhibitor in development, BLU-667 (pralsetinib). Analysts have remarked that the emerging data on the two drugs appear to differentiate them less and less.
Photo: Graeme Robertson, Getty Images
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
CLARIFICATION: An earlier version of this story referred to Eli Lilly & Co. as an owner of Vitrakvi. Although Lilly acquired Loxo Oncology earlier this year, the drug is owned by Bayer, which acquired full commercialization rights in February. The story has been updated.
