Bayer Announces FDA’s First Full Approval of NTRK Inhibitor

FDA has granted full approval to Bayer’s larotrectinib (brand name Vitrakvi), a first-in-class tropomyosin receptor kinase (TRK) inhibitor, for treatment of solid tumors in both adult and pediatric patients. The indication is specifically for those patients who have solid tumors with a neurotrophic receptor tyrosine kinase (NTRK) gene fusion that are without a known acquired resistance mutation, are metastatic, or in cases in which surgical resection may result in severe morbidity. The treatment applies to patients who have no satisfactory alternate treatments or that have progressed following prior treatments (1).

In a press release on April 10, 2025, Bayer said that larotrectinib, which was first granted accelerated approval by FDA in November 2018, demonstrated clinically meaningful and durable responses across a range of NTRK fusion-positive solid tumors, based on data from three multicenter, open-label, single-arm clinical trials (1).

“This first full approval of an NTRK inhibitor by FDA represents the culmination of research and dedication by the Bayer team,” Chandra Goda, executive director and US Vitrakvi brand lead, said in the Bayer press release (1). “We are proud to deliver on our promise for patients with this significant step forward, providing a treatment option for pediatric and adult patients living with NTRK gene fusion-positive cancers."

According to Bayer, TRK fusion cancer occurs when an NTRK gene fuses with another, unrelated gene; this results in an altered TRK protein, called a TRK fusion protein, which becomes constitutively active or overexpressed and can trigger a signaling cascade (1). TRK fusion cancer can occur in any part of the body.

"The full approval of [larotrectinib] by [FDA] is a welcome step forward, solidifying its place as a treatment option for patients with NTRK gene fusion-positive cancers," said Andrea Ferris, president and CEO of the LUNGevity Foundation, in the Bayer release (1). "This milestone not only benefits patients today but also paves the way for further advancements in NTRK gene therapies in the future."

Previously, in 2019 and 2020 respectively, FDA and the European Commission gave approval to Roche’s entrectinib (brand name Rozlytrek) for treating adult and pediatric patients 12 years of age and older with solid tumors that have an NTRK gene fusion without a known acquired resistance mutation, are metastatic, or where surgical resection is likely to result in severe morbidity, and who have progressed following treatment or have no satisfactory alternative therapy (2,3).

In February 2025, EC granted marketing authorization approval to acoramidis (brand name Beyonttra) for treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy, on which Bayer has been collaborating with BridgeBio Pharma since March 2024 (4). For this treatment, Bayer will be responsible for all commercial activity in the EU.

Several weeks earlier, in announcing an acquisition of the clinical-stage radiopharmaceutical contract development and manufacturing organization Evergreen Theragnostics, Massachusetts-based Lantheus Holdings said that in June 2024, it had acquired an RM2—a small peptide that binds with high affinity to the the gastrin-releasing peptide receptor—that was originally developed by Bayer (5).

References

1. Bayer. US FDA Grants Full Approval of Vitrakvi (larotrectinib) for Adult and Pediatric Patients with NTRK Gene Fusion-Positive Solid Tumors. Press Release. April 10, 2025.
2. Roche. FDA Approves Roche’s Rozlytrek (entrectinib) for People with ROS1-Positive, Metastatic Non-Small Cell Lung Cancer and NTRK Gene Fusion-Positive Solid Tumours. Press Release. Aug. 15, 2019.
3. Roche. Rozlytrek, Roche’s First Tumour-Agnostic Therapy, Approved in Europe for People with NTRK Fusion-Positive Solid Tumours and for People with ROS1-Positive Advanced Non-Small Cell Lung Cancer. Press Release. Aug. 2, 2020.
4. BridgeBio Pharma. Beyonttra (acoramidis), The First Near Complete TTR Stabilizer (≥90%), Approved by the European Commission to Treat ATTR-CM. Press Release. Feb. 11, 2025.
5. Lantheus. Lantheus to Acquire Evergreen Theragnostics for Upfront Payment of $250 Million to Drive Strategic Evolution into Fully Integrated Radiopharmaceutical Leader. Press Release. Jan. 28, 2025.