Mirdametinib has shown promising results in patients with neurofibromatosis type 1-associated symptomatic inoperable plexiform neurofibroma (NF1-PN), achieving deep responses regardless of patient demographics. An exploratory analysis of the phase 2b ReNeu trial (NCT03962543) revealed that mirdametinib is effective across a broad spectrum of patient populations.
The analysis, presented at the 2024 Society for Neuro-Oncology Annual Meeting, defined deep responses as an over 50% best reduction in target PN volume. "We didn’t find that there was a particular patient that we could identify who was more likely to be a strong responder. Male or female, young or old—none of these obvious characteristics helped us define who would be a strong responder," said Timothy Gershon, MD, PhD, professor at Emory University School of Medicine.
Duration of Treatment and Response
Notably, the analysis indicated a trend associating longer treatment duration with deeper responses. While the causal relationship remains unclear, it suggests that prolonged therapy may contribute to greater tumor shrinkage. Alternatively, patients achieving larger responses may have remained on therapy longer.
Mirdametinib's Potential and Regulatory Status
Mirdametinib has previously demonstrated a manageable safety profile and the potential for enhancing health-related quality of life (QOL). The FDA granted priority review to mirdametinib in August 2024, with a decision expected by February 28, 2025, underscoring its potential as a valuable therapy for patients with NF1-PN.
