FDA Grants Breakthrough Therapy Designation for Petosemtamab in 1L PD-L1+ Head and Neck Cancer
Merus N.V has announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy designation for petosemtamab in combination with pembrolizumab.
This designation applies to the first-line treatment of adult patients with recurrent or metastatic programmed death-ligand 1 (PD-L1) positive head and neck squamous cell carcinoma (r/m HNSCC) with a combined positive score (CPS) of 1 or higher.
This marks the second BTD designation for petosemtamab. The first was received in May 2024 alongside Fast Track designation in August 2023 for patients with r/m HNSCC whose disease had progressed after platinum-based chemotherapy and anti-programmed cell death protein 1 (anti-PD-1) antibody treatment.
The BTD decision is supported by data from an ongoing phase 1/2 open-label, multicentre trial evaluating the combination of petosemtamab and pembrolizumab in first-line HNSCC cases expressing PD-L1 with CPS≥1 (NCT03526835).
Initial results presented at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting showed a 67% response rate among 24 evaluable patients. The updated interim clinical data provided to the FDA demonstrated the treatment’s efficacy, durability, and safety.
BTD is designed to accelerate the development and review of treatments for serious or life-threatening conditions when preliminary clinical data indicate significant improvement over existing therapies.
It allows for closer FDA guidance, priority review eligibility, and rolling submission of regulatory filings. Following this designation, Merus plans to work closely with the FDA to expedite its Biologics License Application (BLA) submission.
