Neurofibromatosis Type 1 Treatment Landscape: FDA Expands KOSELUGO Approval to Younger Pediatric Patients | DelveInsight's Perspective on Market Impact, Competitive Landscape and Pipeline Therapies

DelveInsight Business Research's analysis underscores the transformative impact of the FDA's September 10, 2025 approval of KOSELUGO (selumetinib) granules and capsules for pediatric patients aged 1 year and older with symptomatic, inoperable plexiform neurofibromas (PN) associated with Neurofibromatosis Type 1 (NF1). This expansion marks the first therapy targeting NF1-PN in this younger age group, offering hope to ~97,000 diagnosed children in the US.
Key NF1 Market Highlights

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The Neurofibromatosis Type 1 treatment market was valued at USD 380 million in 2023, growing robustly through 2034.

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The US captured the largest Neurofibromatosis Type 1 market share at ~USD 230 million in 2023.

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Neurofibromatosis Type 1 Epidemiology: ~97,000 NF1 cases were diagnosed in the US in 2023, with 30-50% of NF1 patients developing PN.

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Leading Neurofibromatosis Type 1 companies include AstraZeneca & Merck, SpringWorks Therapeutics, Healx, Fosun Pharmaceutical, and NFlection Therapeutics.

Access DelveInsight's comprehensive Neurofibromatosis Type 1 Market Report [https://www.delveinsight.com/sample-request/neurofibromatosis-type-1-nf1-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=xpr] to understand the impact of KOSELUGO's expanded pediatric indication and evolving competitive landscape.

Market Impact and Neurofibromatosis Type 1 Patient Population

DelveInsight's NF1 Market Insight, Epidemiology and Market Forecast [https://www.delveinsight.com/report-store/neurofibromatosis-type-1-nf1-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=xpr] report highlights a significant unmet need in pediatric NF1-PN, a condition characterized by benign but often disfiguring nerve sheath tumors with risks of pain, organ dysfunction, and reduced quality of life. The newly approved age expansion addresses a critical treatment gap, allowing intervention at an earlier developmental stage to potentially mitigate long-term morbidity.

Discover Neurofibromatosis Type 1 epidemiology trends, patient population forecasts, and market opportunity assessments [https://www.delveinsight.com/sample-request/neurofibromatosis-type-1-nf1-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=xpr].

KOSELUGO Treatment Approach

KOSELUGO is an oral selective MEK1/2 inhibitor that targets the RAS/MAPK pathway dysregulated by NF1 gene mutations, reducing PN volume and symptomatic burden.

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Granules formulation enables accurate dosing in young children.

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Once-daily administration facilitates adherence in outpatient settings.

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Symptom relief includes reductions in pain, improved motor function, and enhanced quality of life.

"Extending KOSELUGO's indication to children as young as 1 year allows earlier disease modification, which may prevent irreversible complications," said Dr. Hannah Liang, Pediatric Neuro-Oncologist at Children's National Hospital.

KOSELUGO Clinical Validation and Efficacy

FDA approval was based on Pediatric PN Study 4, a multicenter Phase II trial enrolling 120 patients aged 1-3 years with symptomatic, inoperable PN. Key outcomes included:

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35% mean reduction in plexiform neurofibromas volume at 12 months versus baseline (p



This release was published on openPR.