FDA Grants Orphan Drug Designation to PBI-200 for NTRK Fusion–Positive Solid Tumors

The FDA has granted an orphan drug designation to PBI-200 for the treatment of patients with NTRK fusion–positive solid tumors, including primary and metastatic brain tumors. PBI-200, which is an oral, highly potent and selective inhibitor of TRK kinase, was designed by Pyramid Biosciences to overcome a wide range of on-target resistance mutations that have been observed with first-generation TRK inhibitors. “Oncogenic NTRK gene fusions are found in a broad range of cancers. Orphan designation for drugs in development such as PBI-200 are important to ensure continued innovation to address unmet needs for these patients, including primary and metastatic brain tumors,” Brian Lestini, MD, PhD, chief executive officer of Pyramid Biosciences, stated in a press release. Read more >>