Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas

Key Inclusion Criteria:

- Adults ≥ 18 years at enrollment with diagnosis of NF1 with symptomatic, inoperable PN

- At least one inoperable target PN measurable by volumetric MRI analysis

- Chronic target PN pain score documented for minimum period during screening period

- Stable chronic PN pain medication use at enrollment

- Adequate organ and marrow function

Key Exclusion Criteria:

- Confirmed or suspected malignant glioma or MPNST (low grade glioma, including optic
glioma not requiring systemic therapy or radiation therapy are exempt from this
exclusion)

- History of malignancy except for malignancy treated with curative intent with no known
active disease ≥ 5 years before the first dose of study intervention and of low
potential risk for recurrence

- Clinically significant cardiovascular disease, including inherited coronary disease,
acute coronary syndrome within 6 months prior to enrollment, uncontrolled angina,
symptomatic heart failure, cardiomyopathy, severe valvular heart disease, abnormal
LVEF and uncontrolled hypertension

- Ophthalmological findings/conditions including intraocular pressure > 21 mmHg,
RPED/CSR or RVO

- Prior exposure to MEK inhibitors