FDA Grants Breakthrough Status to Novel Therapy for Hard-to-Treat CML
The U.S. Food and Drug Administration (FDA) has named Terns Pharmaceuticals’ investigational oral agent TERN-701 a breakthrough therapy for potential use in certain people with chronic myeloid leukemia (CML), a form of blood cancer[cite: 391]. The FDA awards breakthrough designation to experimental treatments that, based on early trial data, have the potential to meaningfully improve care for people with serious diseases[cite: 394].
Target Profile and Indication
The new designation specifically covers the use of TERN-701 in people with CML whose cancer carries a genetic abnormality called the Philadelphia chromosome but does not have another mutation called T315I[cite: 392]. The treatment candidate is specifically indicated for individuals whose CML is in its chronic phase, the earliest stage of disease where progression is slow and symptoms are minimal, and who have tried at least two other tyrosine kinase inhibitors[cite: 393].
Merck Acquisition and Future Prospects
The new status comes on the heels of a recent announcement that pharma giant Merck will acquire Terns in a cash deal valued at roughly $6.7 billion[cite: 395].
“This Breakthrough Therapy Designation, along with the recent agreement for Merck to acquire Terns, has the potential to accelerate efforts to advance TERN-701 to a pivotal trial and to patients,” Amy Burroughs, CEO of Terns, said in a company press release announcing the new status[cite: 396].
“This is an exciting time for everyone involved in the TERN-701 program,” Burroughs said[cite: 397]. “We are grateful to the investigators, patients and community advocates whose dedication and support have made these advancements possible.” [cite: 398]
