Neurofibromatosis Type 1 Treatment Landscape: FDA Expands KOSELUGO Approval To Younger Pediatric Patients Delveinsight's Perspective On Market Impact, Competitive Landscape And Pipeline Therapies

(MENAFN- GetNews)


"FDA approval of KOSELUGO"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

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

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

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

• 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 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 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 .

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.

• Granules formulation enables accurate dosing in young children.

• Once-daily administration facilitates adherence in outpatient settings.

• 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:

• 35% mean reduction in plexiform neurofibromas volume at 12 months versus baseline (p<0.001).

• 60% of patients achieved ≥20% volume reduction.

• Significant improvements in pain scores and gross motor milestones.

• Tolerable safety profile, with mild gastrointestinal events in 18% of patients.

These data confirmed KOSELUGO's noninferior efficacy in younger patients compared to older cohorts, supporting the label expansion.

Competitive Landscape and Market Positioning

KOSELUGO remains the only FDA-approved therapy for NF1-PN. Emerging competitors include:

• Mirdametinib (SpringWorks Therapeutics), in Phase IIb ReNeu trial for both pediatric and adult PN.

• FCN-159 (Fosun Pharmaceutical), Phase I/II evaluating safety and tolerability.

• PAS-004 (Pasithea Therapeutics), early-stage MEK inhibitor with a 70-hour half-life for once-daily dosing.

KOSELUGO's first-mover advantage, established safety data, and now broadened pediatric label reinforce its market leadership.

Download our analysis of Neurofibromatosis Type 1 pipeline therapies and competitive dynamics shaping the future of NF1-PN treatment.

Emerging NF1 Pipeline Therapies

Key pipeline candidates aiming to address NF1 complexities include:

• HLX-1502 (Healx), a novel modular mitochondrial function modulator entering Phase II.

• NFX-179 (NFlection Therapeutics), early-stage MEK inhibitor for cutaneous NF1 manifestations.

• Cabozantinib (AstraZeneca/Merck), under investigation for symptomatic PN in adults.

These approaches may complement KOSELUGO or provide alternatives for adult patients.

Industry Expert Perspective

“KOSELUGO's pediatric approval expansion is a milestone in NF1 care,” commented Dr. Anjali Kumar, Geneticist at Johns Hopkins University.“Early intervention could alter disease trajectory and improve lifelong outcomes.”

Schedule a consultation with DelveInsight analysts to discuss how KOSELUGO's label expansion influences market growth and treatment strategies.

Looking Forward

DelveInsight analysts project that earlier KOSELUGO intervention may reduce PN-related hospitalizations by 25% and drive NF1 market growth to USD 870 million by 2034, with pediatric cases representing 40% of total revenues. As mirdametinib and other MEK inhibitors advance, competitive dynamics will center on efficacy, safety, and age range, shaping a rapidly evolving NF1 treatment landscape.

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