Neurofibromatosis Type 1 Market Expected to Gain Momentum Through 2034, According to DelveInsight | AstraZeneca, Merck, SpringWorks Therapeutics, Healx, Pasithea Therapeutics, Fosun Pharmaceutical

The Key Neurofibromatosis Type 1 Companies in the market include - AstraZeneca and Merck, SpringWorks Therapeutics, Healx, Pasithea Therapeutics, Fosun Pharmaceutical, NFlection Therapeutics, and others.

DelveInsight's "Neurofibromatosis Type 1 Market Insights, Epidemiology, and Market Forecast-2034 report offers 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.

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Some of the key facts of the Neurofibromatosis Type 1 Market Report:

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The Neurofibromatosis Type 1 market size was valued ~USD 380 million in 2023 and is anticipated to grow with a significant CAGR during the study period (2020-2034)

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In February 2025, SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company specializing in rare diseases and cancer, announced the online publication of data from the pivotal Phase 2b ReNeu trial in the Journal of Clinical Oncology (JCO). The trial evaluated mirdametinib, an investigational MEK inhibitor, in adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN). Previously, findings from the multi-center, single-arm ReNeu trial were presented at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting.

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In November 2024, Alexion, AstraZeneca Rare Disease, and Merck (NYSE: MRK), known as MSD outside the U.S. and Canada, announced positive topline results from the Phase 3 KOMET trial. This global, randomized, double-blind, placebo-controlled multicenter study is the largest of its kind in adults with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PN). The findings revealed that KOSELUGO, an oral and selective MEK inhibitor, achieved a statistically significant and clinically meaningful improvement in objective response rate (ORR), the primary endpoint, compared to placebo in these patients.

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In November 2024, SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company specializing in severe rare diseases and cancer, announced the publication of data from the pivotal Phase 2b ReNeu trial. The study evaluated mirdametinib, an investigational MEK inhibitor, in adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN). The findings are now available online in the Journal of Clinical Oncology (JCO).

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Within the 7MM, the US recorded the highest number of Neurofibromatosis Type 1 cases in 2023, with approximately 97,300 diagnosed prevalent cases. This number is anticipated to rise throughout the forecast period.

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Among the EU4 countries and the UK, Germany reported the highest number of Neurofibromatosis Type 1 cases in 2023, while the UK had the lowest number of cases.

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In the US, approximately 30% of Neurofibromatosis Type 1 PN cases involved children, while adults accounted for about 70% of the cases in 2020.

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In Japan, there were 5,500 operable and 3,900 inoperable cases of Neurofibromatosis Type 1 PN reported in 2023. These numbers are projected to rise over the forecast period.

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Key Neurofibromatosis Type 1 Companies: AstraZeneca and Merck, SpringWorks Therapeutics, Healx, Pasithea Therapeutics, Fosun Pharmaceutical, NFlection Therapeutics, and others

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Key Neurofibromatosis Type 1 Therapies: KOSELUGO (selumetinib), Mirdametinib, HLX-1502, PAS-004*, FCN-159, NFX-179, and others

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The Neurofibromatosis Type 1 market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Neurofibromatosis Type 1 pipeline products will significantly revolutionize the Neurofibromatosis Type 1 market dynamics.

Neurofibromatosis Type 1 Overview

Neurofibromatosis are a group of genetic disorders that cause tumors to form on nerve tissue. There are three types of neurofibromatosis: neurofibromatosis 1 (NF1), neurofibromatosis 2 (NF2) and schwannomatosis.

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Neurofibromatosis Type 1 Epidemiology

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Neurofibromatosis Type 1 Epidemiology Segmentation:

The Neurofibromatosis Type 1 market report proffers epidemiological analysis for the study period 2020-2034 in the 7MM segmented into:

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Total Prevalence of Neurofibromatosis Type 1

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Prevalent Cases of Neurofibromatosis Type 1 by severity

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Gender-specific Prevalence of Neurofibromatosis Type 1

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Diagnosed Cases of Episodic and Chronic Neurofibromatosis Type 1

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Neurofibromatosis Type 1 Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Neurofibromatosis Type 1 market or expected to get launched during the study period. The analysis covers Neurofibromatosis Type 1 market uptake by drugs, patient uptake by therapies, and sales of each drug.

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Neurofibromatosis Type 1 Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Neurofibromatosis Type 1 Therapies and Key Companies

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KOSELUGO (selumetinib): AstraZeneca and Merck

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Mirdametinib: SpringWorks Therapeutics

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HLX-1502: Healx

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PAS-004*: Pasithea Therapeutics

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FCN-159: Fosun Pharmaceutical

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NFX-179: NFlection Therapeutics

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Mirdametinib: SpringWorks Therapeutics

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NFX-179 Gel: NFlection Therapeutics, Inc.

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Scope of the Neurofibromatosis Type 1 Market Report

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Study Period: 2020-2034

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Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

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Key Neurofibromatosis Type 1 Companies: AstraZeneca and Merck, SpringWorks Therapeutics, Healx, Pasithea Therapeutics, Fosun Pharmaceutical, NFlection Therapeutics, and others

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Key Neurofibromatosis Type 1 Therapies: KOSELUGO (selumetinib), Mirdametinib, HLX-1502, PAS-004*, FCN-159, NFX-179, and others

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Neurofibromatosis Type 1 Therapeutic Assessment: Neurofibromatosis Type 1 current marketed and Neurofibromatosis Type 1 emerging therapies

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Neurofibromatosis Type 1 Market Dynamics: Neurofibromatosis Type 1 market drivers and Neurofibromatosis Type 1 market barriers

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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies

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Neurofibromatosis Type 1 Unmet Needs, KOL's views, Analyst's views, Neurofibromatosis Type 1 Market Access and Reimbursement

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Table of Contents

1. Neurofibromatosis Type 1 Market Report Introduction

2. Executive Summary for Neurofibromatosis Type 1

3. SWOT analysis of Neurofibromatosis Type 1

4. Neurofibromatosis Type 1 Patient Share (%) Overview at a Glance

5. Neurofibromatosis Type 1 Market Overview at a Glance

6. Neurofibromatosis Type 1 Disease Background and Overview

7. Neurofibromatosis Type 1 Epidemiology and Patient Population

8. Country-Specific Patient Population of Neurofibromatosis Type 1

9. Neurofibromatosis Type 1 Current Treatment and Medical Practices

10. Neurofibromatosis Type 1 Unmet Needs

11. Neurofibromatosis Type 1 Emerging Therapies

12. Neurofibromatosis Type 1 Market Outlook

13. Country-Wise Neurofibromatosis Type 1 Market Analysis (2020-2034)

14. Neurofibromatosis Type 1 Market Access and Reimbursement of Therapies

15. Neurofibromatosis Type 1 Market Drivers

16. Neurofibromatosis Type 1 Market Barriers

17. Neurofibromatosis Type 1 Appendix

18. Neurofibromatosis Type 1 Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

About DelveInsight

DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.

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