FDA OKs New Next-Gen Pill for Rare Lung Cancer

WebMD

12-06-2025

approved a first-of-its-kind oral pill to treat a rare type of advanced lung cancer that often spreads to the brain.
The new drug, known as taletrectinib but sold as Ibtrozi, is meant for adults with non-small-cell lung cancer (NSCLC) that has spread or worsened and is linked to a faulty ROS1 gene. This targeted therapy offers new hope to about 3,000 people in the U.S. and over 1 million globally who are diagnosed each year with advanced ROS1-positive NSCLC.
NSCLC is the most common form of lung cancer – a leading cause of cancer-related deaths. About 2% of advanced cases are ROS1-positive, which grow faster and are harder to treat. Most patients are diagnosed in their 50s, often without a history of smoking. The cancer often spreads to the brain, seen in 35% at diagnosis and nearly 50% after treatment. This makes treatment especially challenging and highlights the need for better options.
The FDA's decision was based on two clinical studies of people with ROS1-positive NSCLC. One trial involved 113 people who had previously been treated with a drug similar to Ibtrozi, while the other included 157 who had not. About 85% to 90% of those new to treatment responded to Ibtrozi, and at least 63% continued to see benefits for over a year. Among those previously treated, about 52% to 62% improved, with most (up to 83%) continuing to respond for at least six months.
Ibtrozi also helped shrink brain tumors in 73% of people who were new to similar treatments, and in 63% of those who had tried one before, according to a news release by Nuvation Bio, the drug's maker.
"Ibtrozi's durability of response and ability to effectively penetrate the brain, coupled with a well-characterized and manageable safety profile, further addresses these critical needs for patients," said Nathan Pennell, MD, PhD, a professor of medicine at the Cleveland Clinic and an investigator of the clinical trials.
Ibtrozi belongs to a class of medicines called tyrosine kinase inhibitors (TKIs). It blocks the faulty ROS1 protein, helping slow cancer growth and spread. Ibtrozi is designed to reach the brain and keep the disease under control for longer. The FDA recommends 600 mg oral Ibtrozi pills once a day on an empty stomach, with no food two hours before or after taking it. Treatment continues until the cancer worsens or side effects become too serious.
The most common side effects include things like nausea, diarrhea, tiredness, and dizziness. The FDA also warns about more serious risks, like liver and lung problems, heart rhythm issues, muscle pain, and possible harm to an unborn baby. People who use the drug should tell their doctor about all medications they're taking, including over-the-counter ones – especially anything for acid reflux. Women are advised not to breastfeed during treatment and for three weeks afterward. It's also important to protect your skin from the sun while taking the drug and for at least five days after stopping it.