FDA approves selumetinib (Koselugo) for the treatment of children aged from 2 years with neurofibromatosis type 1

Selumetinib, an oral selective MEK inhibitor, is approved specifically for patients who have symptomatic, inoperable plexiform neurofibromas, which are tumours involving nerve sheaths and can grow anywhere in the body, including the face, extremities, and areas around the spine.

SPS commentary:

Neurofibromatosis type 1 is a rare, often disfiguring, progressive condition that typically begins early in life and is characterised by changes in skin colouring, neurologic and skeletal impairments and risk for development of benign and malignant tumours throughout life.

In a phase 2 trial (n=50), 35 patients (70%) with neurofibromatosis type 1 had a confirmed partial response and 28 of these patients had a durable response (lasting ≥1 year).


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Phase II trial