Connecticut Patent of the Month – April 2025

A novel method for treating neurofibromatosis type 1 (NF1) with inoperable plexiform neurofibromas (PN) has been patented, promising improved outcomes for patients. The U.S. Patent No. 12,263,146, titled “Non-linear dosing of mirdametinib,” introduces a tailored dosing regimen based on body surface area (BSA) to enhance treatment efficacy.

Personalized Treatment Approach

Developed by SpringWorks Therapeutics Inc., the patented method administers mirdametinib, a MEK inhibitor, in doses adjusted to the patient’s BSA. For instance, patients with a BSA of 1.5 to 1.74 m² receive 4 mg twice daily, while those with smaller BSAs receive proportionally lower doses. This personalized approach aims to optimize drug exposure and therapeutic response.

Clinical Impact

The dosing strategy is designed to achieve a significant reduction in tumor size, with an objective response rate defined as at least a 20% decrease measured by MRI. Such precision in dosing could lead to better management of NF1-associated PNs, which are often challenging to treat due to their location and growth patterns.

Broader Implications

This innovation not only offers a new avenue for NF1 treatment but also exemplifies the move toward personalized medicine in oncology. By aligning drug dosing with individual patient characteristics, therapies can become more effective and potentially reduce adverse effects.

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