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Neurofibromatosis Type 1 Drugs Market Size Report 2034: Emerging Therapies and Future Growth Opportunities | DelveInsight

07-15-2025 07:39 AM CET | Health & Medicine

Press release from: DelveInsight Business Research LLP

Neurofibromatosis Type 1 Market

Neurofibromatosis Type 1 Market

DelveInsight's "Neurofibromatosis Type 1 Market Insights, Epidemiology, and Market Forecast-2034" report delivers an in-depth understanding of the Neurofibromatosis Type 1, historical and forecasted epidemiology as well as the Neurofibromatosis Type 1 market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

Discover which therapies are expected to grab the Neurofibromatosis Type 1 Market Share @ Neurofibromatosis Type 1 Market Outlook- https://www.delveinsight.com/sample-request/neurofibromatosis-type-1-nf1-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Neurofibromatosis Type 1 (NF1) is a genetic disorder characterized by the development of multiple benign tumors along the nerves and skin, known as neurofibromas. It is one of the most common neurocutaneous disorders and results from mutations in the NF1 gene, which encodes a protein called neurofibromin. This protein is involved in the regulation of cell growth, and its loss leads to uncontrolled cell proliferation and tumor formation.

Key Takeaways from the Neurofibromatosis Type 1 Market Report
• In July 2025, Novartis Pharmaceuticals announced a study to assess long-term effect in pediatric patients treated with dabrafenib and/or trametinib.
• In June 2025, Healx Limited conducted a phase 2 study in 20 participants. The study will assess the tolerability and efficacy of HLX-1502 in participants with NF1 16 years of age or older with progressive and/or symptomatic PN.
• Among the 7MM, the US accounted for the highest number of cases in 2023, with around 97,300 Neurofibromatosis Type 1 diagnosed prevalent cases. These cases are expected to increase during the forecast period.
• Among EU4 and the UK, the highest number of Neurofibromatosis Type 1 cases was found in Germany whereas the UK accounted for the lowest cases in 2023.
• In the US, around 30% of the Neurofibromatosis Type 1 PN cases accounted for children, whereas 70% of the cases accounted for adults in 2020.
• In the Japan, there were 5,500 operable cases and 3,900 inoperable cases of Neurofibromatosis Type 1 PN in 2023. These cases are expected to increase during the forecast period.
• The increase in Neurofibromatosis Type 1 Market Size is a direct consequence of the increasing patient population and anticipated launch of emerging therapies in the 7MM.
• As per DelveInsight analysis, the Neurofibromatosis Type 1 Market is anticipated to witness growth at a considerable CAGR.
• The leading Neurofibromatosis Type 1 Companies such as AstraZeneca, Merck, SpringWorks Therapeutics, Healx, Fosun Pharmaceutical, NFlection Therapeutics, and others.
• Promising Neurofibromatosis Type 1 Pipeline Therapies such as FCN-159, HLX-1502, NFX-179, PAS-004, and others.

Stay ahead in the Neurofibromatosis Type 1 Therapeutics Market with DelveInsight's Strategic Report @ Neurofibromatosis Type 1 Market Outlook- https://www.delveinsight.com/sample-request/neurofibromatosis-type-1-nf1-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

The Neurofibromatosis Type 1 Market is gaining attention due to increased awareness, improved diagnostic capabilities, and advancements in genetic testing. As research deepens the understanding of the molecular basis of the disorder, pharmaceutical companies and biotech firms are investing in the development of novel therapies aimed at managing the symptoms and preventing the progression of the disease.

Neurofibromatosis Type 1 Epidemiology Segmentation in the 7MM
The epidemiology section of Neurofibromatosis Type 1 offers insights into both historical and current patient populations, as well as forecasted trends across seven major countries. This section aids in understanding the factors behind present and projected trends through analysis of various studies and input from key opinion leaders. Additionally, this portion of the market report provides information on the diagnosed patient pool, trends, and underlying assumptions.
• Total Diagnosed Prevalent Cases of Neurofibromatosis Type 1
• Diagnosed Prevalent Cases of Neurofibromatosis Type 1 Manifestations
• Age-specific Diagnosed Prevalent Cases of Neurofibromatosis Type 1
• Severity-specific Diagnosed Prevalent Cases of cNFs
• Age-specific Diagnosed Prevalent Cases of Neurofibromatosis Type 1-PN
• Neurofibromatosis Type 1-PN Cases by Clinical Symptoms
• Neurofibromatosis Type 1-PN Cases Eligible for Surgery

Download the report to understand which factors are driving Neurofibromatosis Type 1 Epidemiology trends @ Neurofibromatosis Type 1 Prevalence- https://www.delveinsight.com/sample-request/neurofibromatosis-type-1-nf1-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Neurofibromatosis Type 1 Marketed Drugs
• KOSELUGO (selumetinib): AstraZeneca and Merck
KOSELUGO is an inhibitor of MEK1/2. KOSELUGO blocks specific enzymes (MEK1 and MEK2), which are involved in stimulating cells to grow. In Neurofibromatosis Type 1, these enzymes are overactive, causing tumor cells to grow in an unregulated way. By blocking these enzymes, KOSELUGO slows down the growth of tumor cells. In April 2020, the FDA approved KOSELUGO for pediatric patients aged 2 and older with symptomatic, inoperable plexiform neurofibromas treatment market associated with Neurofibromatosis Type 1.

Neurofibromatosis Type 1 Emerging Drugs
• Mirdametinib: SpringWorks Therapeutics
Mirdametinib is an investigational oral, allosteric, small-molecule MEK inhibitor in development as a monotherapy treatment for Neurofibromatosis Type 1-PN and Low-grade Glioma (LGG) and as a combination therapy for the treatment of several subsets of biomarker-defined metastatic solid tumors. It was designed to inhibit MEK1 and MEK2, which occupy pivotal positions in the MAPK pathway.

• HLX-1502: Healx
HLX-1502 is a tablet taken orally that works differently than other treatments and offers a new and differentiated investigational treatment option for patients with Neurofibromatosis Type 1. Healx has secured the US FDA clearance for an Investigational New Drug application (IND) to commence a Phase II clinical trial of HLX-1502, targeting adults with Neurofibromatosis Type 1 and inoperable plexiform neurofibroma. Recently, in October 2024, Healx announced that the US FDA granted FTD to HLX-1502 for the treatment of Neurofibromatosis Type 1.

To learn more about Neurofibromatosis Type 1 treatment guidelines, visit @ Neurofibromatosis Type 1 Treatment Market Landscape- https://www.delveinsight.com/sample-request/neurofibromatosis-type-1-nf1-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Neurofibromatosis Type 1 Market Outlook
The Neurofibromatosis Type 1 treatment market landscape has historically been limited to surgical interventions, primarily complete resection or debulking. The tumors' size and location often complicate these approaches, with many being classified as inoperable due to their proximity to vital structures or their invasive nature. Approximately 50% of patients with Neurofibromatosis Type 1-PN face tumors that cannot be safely removed without significant risk of morbidity, necessitating a focus on symptomatic management rather than curative surgery.

Neurofibromatosis Type 1 Drugs Uptake
Surgical options remain the gold standard for Neurofibromatosis Type 1 when feasible. Complete resection is ideal but rarely achievable due to the complex anatomy of these tumors, which often intertwine with nerves and blood vessels. Debulking, or partial removal, may alleviate symptoms but does not guarantee long-term relief, as these tumors frequently regrow. The decision to proceed with surgery is contingent upon a thorough evaluation of the tumor's characteristics and the patient's overall. The approval of KOSELUGO, a selective MEK1/2 inhibitor, marks a pivotal change in the treatment paradigm for pediatric patients with symptomatic, inoperable Neurofibromatosis Type 1-PN in 2020. KOSELUGO is currently the first and only FDA-approved therapy specifically indicated for this condition.

Major Neurofibromatosis Type 1 Companies
AstraZeneca, Merck, SpringWorks Therapeutics, Healx, Fosun Pharmaceutical, NFlection Therapeutics, and others.

Learn more about the FDA-approved drugs for Neurofibromatosis Type 1 @ Drugs for Neurofibromatosis Type 1 Treatment- https://www.delveinsight.com/sample-request/neurofibromatosis-type-1-nf1-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Scope of the Neurofibromatosis Type 1 Market Report
• Coverage- 7MM
• Study Period- 2020-2034
• Neurofibromatosis Type 1 Companies- AstraZeneca, Merck, SpringWorks Therapeutics, Healx, Fosun Pharmaceutical, NFlection Therapeutics, and others.
• Neurofibromatosis Type 1 Pipeline Therapies- FCN-159, HLX-1502, NFX-179, PAS-004, and others.
• Neurofibromatosis Type 1 Market Dynamics: Neurofibromatosis Type 1 Market Drivers and Barriers
• Neurofibromatosis Type 1 Market Access and Reimbursement, Unmet Needs and Future Perspectives

Discover more about Neurofibromatosis Type 1 Drugs in development @ Neurofibromatosis Type 1 Clinical Trials Assessment- https://www.delveinsight.com/sample-request/neurofibromatosis-type-1-nf1-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Table of Contents
1. Key Insights
2. Neurofibromatosis Type 1 Executive Summary
3. Competitive Intelligence Analysis for Neurofibromatosis Type 1
4. Neurofibromatosis Type 1: Market Overview at a Glance
5. Neurofibromatosis Type 1: Disease Background and Overview
6. Patient Journey
7. Neurofibromatosis Type 1 Epidemiology and Patient Population
8. Treatment Algorithm, Current Treatment, and Medical Practices
9. Neurofibromatosis Type 1 Unmet Needs
10. Key Endpoints of Neurofibromatosis Type 1 Treatment
11. Neurofibromatosis Type 1 Marketed Products
12. Neurofibromatosis Type 1 Emerging Therapies
13. Neurofibromatosis Type 1: Seven Major Market Analysis
14. Attribute analysis
15. 7MM: Neurofibromatosis Type 1 Market Outlook
16. Access and Reimbursement Overview of Neurofibromatosis Type 1
17. KOL Views
18. Neurofibromatosis Type 1 Market Drivers
19. Neurofibromatosis Type 1 Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
23. About DelveInsight

About Us

DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.

Company Name: DelveInsight Business Research LLP
Contact Person: Yash Bhardwaj
Email: ybhardwaj@delveinsight.com
Phone: 09650213330
Address: 304 S. Jones Blvd #2432
City: Las Vegas
State: NV
Country: United States
Website: https://www.delveinsight.com/

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